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Check back to this tracker for regulatory updates on marketed drugs, including expansions into new geographies and indications. Welcome to Fierce Pharma's regulatory tracker for the first half of 2025. On this page, we're recording the regulatory progress of in-market products, including expansions into key geographies and new indications. Be sure to come back regularly for the latest updates.UPDATED: Friday, March 14 at 9:25 a.m. ETChina's National Medical Products Administration (NMPA) has approved Innovent's Sycume as a treatment for thyroid eye disease (TED).The drug, also known as teprotumumab N01, becomes the first anti-insulin-like growth factor 1 receptor (IGF-1R) antibody to score approval in the country.In a press release, Innovent said the drug's approval "has ended a 70-year drought of no new treatment option for TED in China."In the U.S., teprotumumab is marketed by Amgen as Tepezza. In Europe, officials have expanded the label for Bristol Myers Squibb's cell therapy Breyanzi.Specifically, the European Commission approved the drug for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy.The approval is based on results from the global phase 2 study called TRANSCEND FL, which enrolled patients with relapsed or refractory indolent non-Hodgkin lymphoma (NHL), including FL. In the third-line-plus treatment setting, 97.1% of patients responded to the drug, and 94.2% of patients achieved a complete response.Before this approval, Breyanzi had scored EU nods to treat certain patients with diffuse large B-cell lymphoma, high grade B-cell lymphoma and primary mediastinal large B-cell lymphoma.UPDATED: Thursday, March 13 at 8:30 a.m. ETKelun-Biotech's TROP 2 ADC sacituzumab tirumotecan (sac-TMT) has scored its second approval in China.The country's National Medical Products Administration (NMPA) approved the drug to treat certain adults with EGFR-positive locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC). The nod covers the drug's use in patients who have progressed after treatment with EGFR-tyrosine kinase inhibitor (TKI) therapy and platinum-based chemotherapy, Kelun said in a press release.With the approval, sac-TMT becomes the first TROP2 ADC approved to treat lung cancer anywhere in the world, according to the company.In a pivotal study, the drug "demonstrated a statistically significant and clinically meaningful improvement" in objective response rate, progression-free survival and overall survival compared with docetaxel, Kelun said. The study tested the drugs in patients with locally advanced or metastatic EGFR-mutant NSCLC who failed after prior treatment with an EGFR-TKI and platinum-based chemo.The approval is sac-TMT's second in the country after the NMPA approved it to treat certain patients with  triple negative breast cancer last year.In 2022, Merck & Co. licensed ex-China rights to the drug.UPDATED: Friday, March 7 at 9:35 a.m. ETThe European Commission (EC) has signed off on Bristol Myers Squibb’s immunotherapy combination of Opdivo and Yervoy to treat first-line patients with advanced or unresectable hepatocellular carcinoma (HCC).The nod was based on Opdivo and Yervoy showing an overall survival edge in the CheckMate-9DW trial over an investigator's choice of oral treatments.The Opdivo-Yervoy combo also faces an FDA decision date of April 21 to treat unresectable HCC. In 2020, the FDA granted accelerated approval to Opdivo-Yervoy to treat second-line advanced HCC.Opdivo and Yervoy were originally approved to treat melanoma, in 2014 and 2011 respectively. In combination, they have been blessed by the FDA in four other cancer types: renal cell carcinoma (2018), colorectal cancer (2018), mesothelioma (2020) and lung cancer (2020).Approximately 90% of liver cancer diagnoses are of the HCC type, BMS said. Liver cancer is the world’s third leading cause of cancer deaths.The FDA has expanded its approval for ALK’s immunotherapy Odactra for house dust mite (HDM) allergies to children ages 5 through 11. The under-the-tongue tablet was originally approved for adults in 2017 and then for adolescents ages 12 to 17 in 2023.UPDATED: Thursday, March 6 at 9:45 a.m. ETARS Pharmaceuticals needle-free epinephrine option, neffy nasal spray, can now be used to treat type 1 allergic reactions including anaphylaxis in children.To win the pediatric nod from the FDA, ARS ran studies proving that pharmacokinetic and pharmacodynamic responses from neffy in both children and adults were consistent with that of standard epinephrine injection products. The label expansion comes after the drug’s August approval as the first major innovation in epinephrine delivery in more than 35 years. Needle-free delivery is especially significant in the pediatric population as many children and caregivers fear needle-based auto-injectors, standing to delay lifesaving treatment.Human factor studies also show that children as young as 10 can use the nasal spray effectively and that untrained individuals, such as babysitters or teachers, can also correctly administer the drug. In the U.S., nearly 4 out of every 10 epinephrine prescriptions are for children under the age of 18 and some one in 13 children have severe food allergies, with more than 40% having experienced severe reactions, according to ARS. UPDATED: Wednesday, March 5 at 10:10 a.m. ETRoche's supplemental biologics application for Gazyva to treat lupus nephritis has been accepted for review by the FDA, the company said in a Wednesday press release.The filing leverages results from the phase 3 REGENCY study, which showed that 46.4% of patients with lupus nephritis who received Gazyva plus standard care achieved a complete renal response after 76 weeks. For patients who received standard care alone, the number was 33.1%, according to study results published last month.First approved in 2013, Gazyva is already cleared to treat chronic lymphocytic leukemia and follicular lymphoma in certain situations.The FDA is expected to make a decision on the lupus nephritis filing by October, according to Roche.UPDATED: Monday, March 3 at 4:30 p.m. ETDespite a reconsideration after a prior rejection, Australian drug regulators ultimately passed on Eisai's BioArtic-partnered Alzheimer’s disease treatment Leqembi. The Therapeutic Goods Administration (TGA) of Australia first decided against approval for patients with early Alzheimer’s disease in October. Eisai then requested reconsideration of the decision in December, proposing the same narrow indication given to the drug by the Medicines and Healthcare products Regulatory Agency (MHRA) and European Medicines Agency (EMA). Those approvals stipulate that Leqembi can only be used in patients with one or no copies of the ApoE4 gene due to safety concerns linking two copies of the gene to increased risks of brain swelling and bleeding, a side effect known as Amyloid Related Imaging Abnormalities (ARIAs).However, the TGA did not agree with the safety profile of Leqembi in ApoE4 heterozygotes, or those with one copy of the ApoE4 gene. Instead, the regulator offered an alternative narrow indication covering only ApoE4 noncarriers. Eisai floated other indications instead, including one that would keep the ApoE4 noncarrier and heterozygote patient populations but require heterozygotes to receive treatment in specialist centers with expert physician supervision. Still, the TGA rejected those options.BioArtic, Eisai’s long-time research partner that originally developed the antibody that became Leqembi, said it’s “very disappointed” by TGA’s decision, CEO Gunilla Osswald said in a statement. Eisai said it remains committed to ensuring eligible Australians can access the treatment and is “exploring options” to achieve this including a potential review by Australia’s Administrative Review Tribunal. UPDATED: Friday, Feb. 28 at 10:30 a.m. ETThe CDC said it's investigating five hospitalizations related to heart and nervous system events following vaccination with Valneva's Ixchiq among people 65 years and older. Ixchiq became the first FDA-approved chikungunya vaccine in November 2023. The CDC currently recommends the live-attenuated shot to be used for adults traveling to a country or territory where there is a chikungunya outbreak. Based on information from the CDC, at least four of the five individuals had also received other vaccines, and most of them had significant underlying medical conditions, a Valneva spokesperson said in a statement to Fierce Pharma."Valneva has not identified any safety signal concerns through post-marketing safety monitoring, including periodic safety reports and routine signal detection activities, and Ixchiq’s safety profile remains consistent with clinical trial data, unchanged and positive," the spokesperson said.The CDC said findings from its investigation will be discussed with experts at an Advisory Committee on Immunization Practices (ACIP) meeting. This year's first ACIP meeting, which was supposed to take place this week, was postponed with no new time provided by the CDC.The U.K. has approved Moderna's respiratory syncytial virus (RSV) vaccine mRESVIA to protect patients ages 60 and over against lower respiratory tract disease caused by the virus.In a study conducted in about 37,000 older adults, people who received mRESVIA had a 79% reduction in their risk of getting lower respiratory tract disease caused by RSV, compared with those who received placebo, around four months after vaccination.The shot will be manufactured at the Moderna Innovation and Technology Centre in Oxfordshire, which will be fully operational later this year, CEO Stéphane Bancel said in a statement.As is the case in the U.S., Moderna's mRNA shot will compete with GSK's Arexvy and Pfizer's Abrysvo in the U.K. as well.UPDATED: Thursday, Feb. 27 at 8:32 a.m. ETAfter being turned away by the FDA nearly one year ago, Regeneron this week said that the U.S. agency has accepted for review its biologics license application for bispecific antibody odronextamab.The FDA is expected to act on the application by July 30, Regeneron said in a Wednesday press release.In March of 2024, the agency rejected an accelerated approval filing for the drug based on the timeline of Regeneron's ongoing confirmatory trials. That decision spurred some confusion at Regeneron—and beyond—about a new requirement from the agency that confirmatory trials be "well underway" by the time it awards accelerated approvals.Early this year, the FDA sought to clear up the issue with new guidance on its expectations around accelerated approvals and confirmatory trial timelines.Nevertheless, Regeneron's review for the drug to treat patients with relapsed/refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy now appears back on track. In its latest press release, Regeneron said the resubmission "follows the achievement of an FDA-mandated enrollment target for the Phase 3 confirmatory trial." As the company notes, the enrollment target was the "sole approvability issue identified by the FDA" in its last submission.In Europe, regulators last year approved the drug for patients with R/R FL and R/R diffuse large B-cell lymphoma after two or more lines of systemic therapy. The medicine carries the brand name Ordspono in the region.In a phase 2 study, 80% of patients with FL who were treated with odronextamab responded to the therapy, with 74% achieving a complete response.UPDATED: Tuesday, Feb. 25 at 9:30 a.m. ETMerck's Keytruda, already boasting some 40 FDA approvals, has picked up a priority review in a proposed new use.The company's filing for the drug as part of a perioperative regimen for patients with resectable locally advanced head and neck squamous cell carcinoma has been accepted for a speedy review, Merck said on Tuesday.The filing seeks approval for Keytruda as a neoadjuvant treatment prior to surgery, followed by its administration as an adjuvant treatment in combination with standard of care radiotherapy—with or without cisplatin—after surgery. Afterward, the company's filing proposes Keytruda being used as a single agent.Merck's application leverages results from the KEYNOTE-689 trial, which showed that the perioperative regimen significantly reduced the risk of disease recurrence, worsening or death in certain patients with resected HNSCC compared with traditional postsurgical radiotherapy alone.The FDA is expected to act on the application by June 23.In Japan, Sanofi's Sarclisa has picked up an approval to treat newly diagnosed patients with multiple myeloma.Japan's Ministry of Health, Labour and Welfare (MHLW) approved the drug as part of a combination with bortezomib, lenalidomide, and dexamethasone (VRd) in the patient group based on results from the phase 3 IMROZ trial. The study showed that Sarclisa's addition to VRd significantly improved progression-free survival compared with VRd alone in patients with newly diagnosed, transplant-ineligible multiple myeloma.Sarclisa launched in 2020 in Japan and is approved as a part of four different regimens in the country, Sanofi noted in a press release.UPDATED: Monday, Feb. 24 at 10:52 a.m. ETGilead's application for twice-yearly lenacapavir has been accepted by the European Medicines Agency for accelerated review as an HIV pre-exposure prophylaxis (PrEP) option. Simultaneously, the drug will be assessed under EU-Medicines for all, which can be used to facilitate expedited review processes in countries outside the EU, including in low- and lower-middle-income countries. The FDA has put lenacapavir's PrEP application under priority review last week with a target decision date set for June 19, 2025.Several companies have launched their biosimilar products referencing Johnson & Johnson's star inflammatory disease drug Stelara in the U.S., following Amgen's January launch of Wezlana as the first biosimilar referencing the star IL-12/23 inhibitor. Teva and Alvotech just rolled out Selarsdi covering all indications and presentations of the original Stelara. Teva can start to claim interchangeability after an exclusivity period for Wezlana ends on April 30. Sandoz announced the launch of its Samsung Bioepis-partnered Pyzchiva, which the Swiss company views as a key driver in its global growth strategy. Sandoz touts Pyzchiva's extended stability, including the ability to be re-refrigerate, as an advantage over the original Stelara.In addition, Biocon Biologics has also entered the U.S. Stelara biosimilar market with Yesintek. It marks the Biocon subsidiary's "first product launch in the United States since becoming a fully integrated global biosimilars organization," CEO Shreehas Tambe said in a release. The FDA has granted priority review to Bristol Myers Squibb's application for Opdivo and Yervoy in first-line unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (mCRC). The goal date is June 23, 2025.The filing was based on data from the phase 3 CheckMate-8HW trial. The combo lowered the risk of disease progression or death by 38% versus Opdivo alone in MSI-H/dMMR mCRC, according to an update of the study presented in January.The FDA has updated the label of Indivior's Sublocade for severe opioid use disorder. The new label allows healthcare providers to start treatment with Sublocade after a single dose of transmucosal buprenorphine and a one-hour observation period. This rapid initiation "may lessen some of the practical obstacles to treatment induction," according to Indivior.In addition, the FDA has approved additional injection sites for Sublocade, an extended-release formulation of buprenorphine. The drug can now be administered in the abdomen, thigh, buttock or back of the upper arm.UPDATED: Friday, Feb. 21 at 9:30 a.m. ETGilead's seladelpar has secured a conditional marketing nod in Europe to treat adults with primary biliary cholangitis (PBC).The approval covers seladelpar's use in combination with ursodeoxycholic acid (UDCA) for those who have an inadequate response to UDCA alone, or as a monotherapy for people who can't tolerate UDCA.In the U.S., seladelpar picked up FDA approval last year and is branded as Livdelzi.A chronic autoimmune disease, PCB affects roughly 22 out of 100,000 people in Europe. The disease is more common in women and causes liver damage that can progress to liver failure if untreated. Symptoms include chronic itch and fatigue, and there is no cure for the disease.The approval is largely based on results from the phase 3 Response study, which showed that roughly three times as many patients on seladelpar achieved a composite biochemical response than those on placebo. The conditional approval will be contingent on positive results from future confirmatory studies.In Japan, authorities have approved CSL's subcutaneous Andembry for the prevention of acute attacks of hereditary angioedema (HAE).The drug offers patients the first pre-filled pen presentation for monthly subcutaneous administration for long-term prophylaxis of HAE, CSL said in a press release.The Japanese nod adds to the drug's other approvals in Europe, Australia and the U.K.UPDATED: Thursday, Feb. 20 at 10:00 a.m. ETBristol Myers Squibb's Breyanzi has won reimbursement backing from England's National Institute for Health and Care Excellence (NICE) in second-line large B-cell lymphoma, becoming the first CAR-T therapy to be recommended by the agency for this cancer type. The endorsement comes after NICE had originally rejected Breyanzi in October 2024 during its draft assessment. The drug cost watchdog changed its mind after BMS "offered an improved commercial arrangement" to the NHS based on its 297,000 pound sterling (about $375,000) list price per person, according to a press release.About 600 people in England could benefit from the therapy per year, according to NICE, which called the one-time CAR-T treatment "a significant advance in lymphoma care." In another reversal, NICE has also changed a previous rejection and signed off on UCB's Fintepla as an add-on treatment for seizures associated with Lennox-Gastaut syndrome (LGS) in people ages 2 years and above. The drug already has coverage under NHS for treating seizures from Dravet syndrome.LGS is a rare but severely debilitating form of epilepsy that begins in early childhood. By NICE's estimate, about 1,400 patients live with the disease in its jurisdiction.NICE changed its mind after UCB offered "an improved discount" to the drug's list price, the agency noted. Using a cost comparison approach, UCB convinced NICE's drug reviewers that Fintepla provides similar or greater health benefits compared to cannabidiol plus clobazam—which is already covered for LGS in the NHS—at a similar or lower cost.  The drug is covered only if the frequency of drop seizures is checked every six months, at which point the frequency is reduced by at least 30% compared with before starting treatment. UPDATED: Tuesday, Feb. 18 at 11:04 a.m. ETMerck’s Welireg snagged its first two approvals in Europe, with the nods covering certain patients with von Hippel-Lidau (VHL) disease and advanced clear renal cell carcinoma (RCC).The European Commission specifically approved the medicine as a treatment for those with VHL disease who have associated, localized RCC, central nervous system hemangioblastomas or pancreatic neuroendocrine tumors. The drug is approved in cases when localized procedures are unsuitable, making Welireg the first available treatment for VHL-associated tumors in Europe. The med also won a nod for adult patients with advanced clear cell RCC that has progressed following two or more lines of therapy, including a PD-1/L1 and at least two VEGF-targeted therapies.The European nods, which are conditional authorizations, follow similar approvals in the U.S. Merck will make the drug commercially available individual European countries after the complication of national reimbursement procedures, among other launch timing factors, the company said.Gilead’s twice-yearly pre-exposure prophylaxis (PrEP) candidate lenacapavir is on the FDA’s review table with a decision date of June 19.The drug, which is currently approved as Sunlenca for multi-drug resistant HIV, would be the first twice-yearly PrEP option on the market. The company’s FDA bid is the first step of its detailed global launch and access strategy.Gilead’s lenacapivir application is backed by two phase 3 trials, one of which showed an impressive 100% risk reduction. The company describes the med as a “foundation” for potential future HIV therapies, it said in a release.Sanofi and Regeneron’s immunology powerhouse Dupixent is aiming to secure a new use in chronic skin disease bullous pemphigoid (BP) with an FDA decision expected by June 20.If approved, the med would be the only targeted BP treatment available in the U.S. The disease is hallmarked by intense itch and blisters and impacts a population of around 27,000 adults, typically elderly adults, whose condition is not controlled by systemic corticosteroids.  The companies’ FDA bid has been granted priority review and is based on a study of 106 adults with moderate-to-severe BP. In the trial, five times more patients who received Dupixent achieved sustained disease remission compared to those on placebo, according to Sanofi. UPDATED: Friday, Feb. 14 at 9:20 a.m. ETCSL's hereditary angioedema (HAE) therapy Andembry has secured approval from the European Commission.The drug is the first once-monthly treatment targeting factor XIIa to prevent HAE attacks in patients 12 and older. In a phase 3 trial, the drug significantly reduced investigator-confirmed HAE attacks per month versus placebo, according to results published in The Lancet in April 2023.Last month, regulators in Australia and the U.K. signed off on the medicine."Andembry, CSL's first approved recombinant monoclonal antibody discovered and developed entirely by CSL, underscores our more than 40-year legacy in HAE research and treatment optimization and our decades-long journey to bring this innovation to patients," Bill Mezzanotte, M.D., CSL's head of R&D, said in a press release.Now, CSL plans to engage in access and reimbursement negotiations with national authorities across Europe as the company progresses with the rollout.Also in Europe, officials signed off on CSL and Arcturus Therapeutics' self-amplifying mRNA COVID-19 vaccine, Kostaive, for use in individuals ages 18 and older.The approval is based on results from an integrated phase 1/2/3 study and late-stage booster trials, the partners said in a press release. Late last year, the European Medicines Agency recommended the vaccine for approval.Besides Europe, the vaccine is approved in Japan.Galderma's efforts to expand the market for Nemluvio are bearing more fruit.In Europe, officials approved the subcutaneous drug for the treatment of moderate-to-severe atopic dermatitis in patients ages 12 and older. In addition, the drug scored an endorsement to treat prurigo nodularis in adults.In the late-stage Arcadia and Olympia clinical trial programs, the drug "significantly improved itch, skin lesions and sleep disturbance, in patients with moderate-to-severe atopic dermatitis and adults with prurigo nodularis, respectively," Galderma said in a Friday press release.The drug is also approved in the U.S. and is under review in Canada, Brazil, South Korea and other countries.China has approved its 22nd PD-1/L1 product. The approval went to SinoCellTech's finotonlimab for its use alongside chemo in first-line head and neck cancer. In a China phase 3 trial, the combo reduced the risk of death by 27% versus chemo alone, extending the median overall survival by 3.6 months to 14.1 months, according to results published in Nature Medicine.The go-ahead marks the first for a PD-1/L1 inhibitor that covers all head and neck cancer patients in China. In the country, Merck & Co.'s Keytruda is allowed as a monotherapy only in PD-L1-positive disease, SinoCellTech noted in a securities filing (Chinese). UPDATED: Wednesday, Feb. 12 at 10:20 a.m. ETAcoramidis, the transthyretin amyloid cardiomyopathy (ATTR-CM) drug developed by BridgeBio and licensed by Bayer in Europe, has been approved by the European Commission. Bayer plans to make the transthyretin stabilizer available in Europe under the brand name Beyonttra in the first half of this year.The approval is based on results from the phase 3 ATTRibute-CM study trial showing that Beyonttra significantly reduced the composite endpoint of all-cause mortality and cardiovascular-related hospitalizations compared with placebo. During the open-label extension phase of the study, Beyonttra demonstrated a relative risk reduction in deaths of 34% after 42 months.Beyonttra's launch is one of Bayer's “must-win battles” as the German company navigates the loss of exclusivity of its top-selling drug, blood thinner Xarelto, Stefan Oelrich, head of Bayer’s pharma division, said at the J.P. Morgan Healthcare Conference in January.Travere Therapeutics said it has aligned with the FDA on its plan to submit Filspari for traditional approval in focal segmental glomerulosclerosis (FSGS), a rare kidney disease. The move comes after a Filspari phase 3 trial in that indication failed on its primary endpoint of eGFR slope, a measurement of kidney disease progression. It also comes as findings from a collaborative global project called ARASOL linked a decrease in proteinuria over 24 months to a reduction in the risk of kidney failure in FSGS. Filspari has shown a benefit in lowering proteinuria. Travere plans to file this application around the end of March using existing data from the phase 3 DUPLEX and phase 2 DUET studies. The company will request a priority review, which Leerink Partners analysts believe is likely to be granted as there are no approved therapies for FSGS. Given that the understanding around proteinuria as a potential full approval endpoint is new, Leerink analysts said it would not be surprising if the FDA wanted to discuss the results during an advisory committee meeting.An approval in FSGS would be "transformational" for Filspari and Travere, Leerink said in a Tuesday note, because it could give the drug $1.1 billion in peak sales across the U.S. and EU based on a "relatively conservative peak penetration" of less than 20%. For Filspari sales, the FSGS indication could eventually surpass IgA nephropathy, for which Filspari is currently approved and is starting to face more competition, according to Leerink Partners.UPDATED: Monday, Feb. 10 at 9:45 a.m. ETBristol Myers Squibb’s cancer combo Opdivo plus Yervoy has scored an approval recommendation in the E.U., thanks to an endorsement from the EMA’s Committee for Medicinal Products for Human Use (CHMP) as a first-line treatment in adults with unresectable or advanced hepatocellular carcinoma.With the thumbs up from CHMP, the Opvido-Yervoy combo now moves forward to the European Commission (EC) for a final approval decision.The CHMP based its green light on results from the combo’s phase 3 CheckMate -9DW trial, in which patients on Opdivo and Yervoy charted a median overall survival of 23.7 months, compared to 20.6 months in patients who received an alternative regimen of Lenvima (lenvatinib) or sorafenib.BMS’ cancer combo is up for a similar approval in the U.S., with the FDA scheduled to make its decision on a potential first-line unresectable HCC nod by April 21.The drugmaker also won a CHMP endorsement for its CAR-T therapy Breyanzi to treat adults with relapsed or refractory follicular lymphoma (FL) who’ve previously tried two or more systemic therapies.BMS used data from its phase 2 TRANSCEND FL study to support the recommendation. In the trial, Breyanzi charted an overall response rate of 97.1% and a complete response rate of 94.2%, which was sufficient to meet the study’s primary and secondary endpoints, BMS said in a release.Breyanzi already carries multiple oncology approvals in Europe, including in relapsed or refractory diffuse large B-cell lymphoma and high grade B-cell lymphoma.Late last month, the FDA gave a thumbs up to German drugmaker medac’s alkylating agent Grafapex, also known as treosulfan, alongside the antimetabolite medicine fludarabine, as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adults and kids ages one and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).Alkylating agents cause breakage of DNA strand to help thwart the ability of cancer cells to multiply.Medac’s approval was backed up by a study looking primarily at overall survival in 570 adults ages 18 to 70 years old with AML or MDS who were randomized to receive either Grafapex or busulfan with fludarabine as a preparative regimen for allogeneic transplant. UPDATED: Wednesday, Feb. 5 at 9:05 a.m. ETNovartis' Kisqali has secured a label expansion from the U.K.'s Medicines and Healthcare products Regulatory Agency (MHRA).The new approval covers Kisqali's use in combination with an aromatase inhibitor (AI) in patients with HR+/HER2- early breast cancer at high risk of recurrence. Previously, the drug was approved by the MHRA to treat advanced breast cancer, according to a Feb. 4 press release from Novartis.The latest approval is based on results from the phase 3 NATALEE trial, which showed that the addition of Kisqali to treatment with an AI cut the risk of invasive disease, recurrence, or death compared to AI treatment alone in patients with HR+/HER2- early breast cancer.Last fall, the drug picked up an FDA nod in patients with HR+/HER2- early breast cancer based on the NATALEE study results.Vaccine specialist Valneva has picked up an approval from the MHRA, as well.The agency approved the company's chikungunya vaccine, Ixchiq, for use in people 18 years of age and older. Late-stage trial data show the vaccine "induces a rapid and strong immune response," according to a press release from the company.Valneva manufactures the shot at its facility in Livingston, Scotland.Previously, the vaccine secured approvals in the United States, Canada and Europe. UPDATED: Friday, Jan. 31 at 9:35 a.m. ETVertex's groundbreaking launch of Casgevy keeps racking up more milestones around the globe.Friday, the company said it has reached a reimbursement agreement with NHS England that'll allow eligible patients with sickle cell disease access to the CRISPR/Cas9 gene-edited therapy. Along with the NHS England agreement, the National Institute for Health and Care Excellence (NICE) has signed off on the drug's use, Vertex said in a press release.The reimbursement deal follows a similar agreement, unveiled last summer, for the drug in patients with transfusion-dependent beta thalassemia.“We are pleased to have reached this new agreement that ensures both eligible SCD and TDT patients can now be treated with Casgevy, recognizing the value a one-time treatment can provide to patients, their families and the healthcare system," Vertex International senior vice president Ludovic Fenaux said in a statement.In its approval bid for Fabhalta in C3 glomerulopathy, Novartis will no longer face an FDA advisory committee meeting, CEO Vas Narasimhan said on Friday.The advisory committee meeting had been scheduled for next month but will no longer take place.Speaking with members of the media on Friday, Narasimhan said the "FDA no longer deemed it necessary to hold an advisory committee meeting.""We think it's based on, you know, the overall data package we've been able to generate, and the additional follow on data that we've provided the agency," the CEO continued. So we look forward to now bringing forward that medicine as a third indication approval for Fabhalta."Amid Europe's ongoing regulatory review of Eisai and Biogen's Alzheimer's disease medicine Leqembi, the European Commission (EC) has flagged new safety information for review.In November, Europe's Committee for Medicinal Products for Human Use (CHMP) recommended market approval of the drug. Since then, EC has asked the committee to "consider information on the safety of lecanemab that became available after the adoption of the CHMP opinion," the partners said in a Friday press release. The companies didn't reveal the nature of the new safety information flagged by the EC. CHMP will dig into the new info during a meeting next month, according to the release."We believe that the EC’s requests can be addressed with existing information and will be evaluated by the CHMP because of the clear and sufficient information available," the partners said in the release. "We will continue to work closely with the authorities toward approval in the EU."UPDATED: Monday, Jan. 27 at 10:43 a.m. ETMerck’s Welireg is up for a potential FDA label expansion by May 26. The agency granted a priority review to the company's bid to expand the drug's label into advanced, unresectable, or metastatic pheochromocytoma and paraganglioma (PPGL), which would add to currently approved indications in advanced renal cell carcinoma and von Hippel-Lindau disease.Welireg generated $139 million in sales during 2024’s third quarter and is the only approved HIF-2α inhibitor in the U.S.Merck’s application in PPGL is based on objective response rate and duration of response rate data from the phase 2 LITESPARK-015 trial, which enrolled 322 patients and will be presented at an upcoming medical meeting, according to the company. Up to 2,000 new cases of the tumors are diagnosed yearly in the U.S.Merck is evaluating Welireg across other rare oncologic diseases both on its own and in combination with other therapies. The FDA is evaluating Alvotech and Teva’s proposed biosimilar to Johnson & Johnson’s Simponi and Simponi Aria.Alvotech, which is responsible for development and manufacturing under its longstanding collaboration with Teva, announced positive top line results from its confirmatory trial of the Simponi copycat back in April. The partners expect the FDA to complete its review process in the fourth quarter of 2025. Simponi was first approved in 2009 and treats a variety of inflammatory conditions including rheumatoid arthritis, psoriatic arthritis and ankylosing spondylitis, among others. Simponi is an injected drug, while Simponi Aria is the infused version. The franchise picked up $2.19 billion for J&J in 2023 sales.Alvotech and Teva's product is the first biosimilar to the blockbuster arthritis med to be accepted for review.UPDATED: Thursday, Jan. 23 at 10:00 a.m. ETBiogen said its applications for a high-dose version of the spinal muscular atrophy med Spinraza have been accepted for review by the FDA and the European Medicines Agency. The new regimen comprises a more rapid loading regimen of two 50 mg doses given two weeks apart and a maintenance regimen given at 28 mg every four months. Currently, the drug's FDA-approved loading regimen features three doses given 14 days apart, plus a fourth dose 30 days after the third. The drug is currently approved in 12 mg doses.The high-dose applications are based on results from the phase 2/3 Devote trial, which favored the high-dose version versus an external sham control and the currently available dose on the measure of patients' reduction in plasma neurofilament light chain (NfL), a marker for neurodegeneration.The higher dose regimen also reduced the risk of death or permanent ventilation by 68% relative to sham and 30% compared with the 12 mg regimen.The high-dose Spinraza is Biogen's answer to competitors such as Novartis' gene therapy Zolgensma.Amneal has won FDA approvals for generics referencing AbbVie's Alzheimer's combo drug Namzaric and Novartis' cancer therapy Afinitor Disperz. Amneal has launched the Namzaric generic with 180-day market exclusivity.Separately, Amneal said it has won tentative approval from the FDA for a generic to Bausch Health's star irritable bowel syndrome med Xifaxan. Bausch Health has previously reached settlement agreements with Teva, Sun Pharma and Sandoz, pushing their generic entry dates into 2028 or upon an earlier launch of a rival generic product. Xifaxan is one of 15 drugs up for the second cycle of Medicare price negotiations under the Inflation Reduction Act. UPDATED: Tuesday, Jan. 21 at 8:57 a.m. ETThe European Commission has approved Johnson & Johnson's combination of Lazcluze and Rybrevant for the first-line treatment of certain adults with advanced non-small cell lung cancer (NSCLC), the company said in a press release.Specifically, the combo is approved in Europe for patients with epidermal growth factor receptor (EGFR) exon 19 deletions (ex19del) or exon 21 L858R (L858R) substitution mutations.In approving the use, EU regulators reviewed results from the phase 3 MARIPOSA study. The trial showed that the combination reduced the risk of disease progression or death by 30% compared with AstraZeneca's Tagrisso in patients with newly diagnosed, EGFR-mutant non-small cell lung cancer. The results yielded a U.S. approval for the J&J combination last summer. Earlier this month, J&J touted an overall survival win from the study. At the time, a company executive heralded the regimen as "a new standard of care for patients with EGFR non-small cell lung cancer."Also in Europe, officials have endorsed GSK's Jemperli to treat all adult patients with primary advanced or recurrent endometrial cancer, including those with mismatch repair proficient (MMRp)/microsatellite stable (MSS) tumors. MMRp and MSS cases represent approximately 75% of patients diagnosed with endometrial cancer, GSK said in a press release.The approval covers Jemperli's use in combination with chemotherapy.Late last year, Europe's Committee for Medicinal Products for Human Use (CHMP) recommended the label expansion.In England, the National Institute for Health and Care Excellence (NICE) recommended AstraZeneca's Tagrisso for treating people with EGFR mutation-positive non-small-cell lung cancer (NSCLC) after surgery.The recommendation means that hundreds of patients will benefit from routine access to the drug, which is taken daily alongside chemotherapy, according to a notice from the Roy Castle Lung Cancer Foundation.Previously, EGFR mutation-positive NSCLC patients relied on chemotherapy to prevent cancer recurrence after surgery, according to the foundation.“I am pleased we have been able to recommend that this targeted treatment for a specific gene mutation of lung cancer will be routinely available on the NHS," NICE's director of medicines evaluation, Helen Knight, said in a statement.UPDATED: Thursday, Jan. 16 at 10:25 a.m. ETEli Lilly's Omvoh has picked up its second major FDA nod. After the IL-23 inhibitor's approval in 2023 to treat ulcerative colitis, the agency has now signed off on Omvoh to treat Crohn's disease. Specifically, the FDA approved Omvoh to treat moderately to severely active Crohn's disease in adults.In a phase 3 study, 53% of patients treated with Omvoh achieved clinical remission after a year of treatment. That number compared with 36% for those on placebo.In addition, 46% of patients treated with Omvoh had visible healing of the intestinal lining after a year. In the placebo group, the number was 23%.The trial enrolled patients with moderately to severely active Crohn's disease who had experienced an inadequate response, loss of response or who could not tolerate corticosteroids, immunomodulators and/or biologics.With the FDA nod, Lilly's drug will go up against some heavy hitters in the Crohn's space, including Johnson & Johnson's Stelara and AbbVie's duo of Skyrizi and Rinvoq.UPDATED: Wednesday, Jan. 15 at 8:12 a.m. ETSanofi's Sarclisa has gained approval from China's National Medical Products Administration (NMPA) to treat adults with multiple myeloma who've received at least one prior line of therapy, including lenalidomide and a proteasome inhibitor.The approval covers Sarclisa's use in combination with pomalidomide and dexamethasone (Pd).Regulators in China endorsed the drug based on results from the phase 3 ICARIA-MM study, as well as bridging data from a China-based study, Sanofi said in a press release. In ICARIA-MM, the addition of Sarclisa to Pd resulted in a 40% reduction in patients' risk of disease progression or death. "We look forward to continuing to build strong partnerships with the medical community, local companies, and authorities in China as we work to bring more innovative treatments to patients," Sanofi's head of oncology, Olivier Nataf, said in a statement.As Biogen and Eisai work to grow Leqembi in Alzheimer's disease, the FDA has accepted a biologics license application for the drug's subcutaneous maintenance dosing option. With a potential approval, Leqembi would become the first anti-amyloid therapy to offer at-home maintenance dosing.The FDA is set to make a decision on the filing by August 31, according to a Biogen press release.UPDATED: Thursday, Jan. 9 at 11:15 a.m. ETEngland’s National Institute for Health and Care Excellence (NICE) recommended AstraZeneca’s Lynparza for use in the region’s National Health Service, clearing the way for around 1,200 eligible patients to reap the benefits of the treatment.The recommendation covers Lynparza’s use in patients with HER2-negative, locally advanced or metastatic breast cancer who have BRCA1 or BRCA2 mutations and have had chemotherapy. NICE based its decision in part on results from the company’s OlympiAD phase 3 study, which proved that the drug could cut the risk of disease worsening or death by 42%.NICE had originally begun its assessment of the treatment in that indication in 2018, but ended it citing a lack of evidence submitted by the company. This time around, NICE assessed the med using a faster cost comparison process that compared Lynparza to Pfizer’s Talzenna in benefits and cost. Stallergenes Greer’s peanut allergy treatment Palforzia scored a label expansion in Europe, extending the drug’s use to toddlers aged 1 through 3 years old.Palforzia is now cleared for use in those aged 1 to 17 years old and is the only approved oral immunotherapy in the U.S. and Europe for toddlers with a confirmed peanut allergy diagnosis. The drug is meant to gradually increase the body’s ability to tolerate small amounts of peanuts through carefully controlled initial dose escalation and up-dosing.The European Commission based its nod on the company’s POSEIDON phase 3 study, which was a double-blind, placebo-controlled food challenge after six months of treatment. Agios Pharmaceuticals’ Pyrukynd is up for an FDA decision in non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia on Sept. 7.The company filed its bid for approval last month after finding success in two phase 3 trials assessing the drug in the non-transfusion-dependent and transfusion-dependent populations. Thalassemia is a rare, inherited blood disorder with limited treatment options. The disease is hallmarked by the abnormal production of hemoglobin, which causes chronic anemia and can lead to other complications.Pyruknd won its first approval in 2022 for pyruvate kinase (PK) deficiency, marking the first drug approved to treat the disease. Merck’s Gardasil HPV vaccine won approval from the National Medical Products Administration of China for males aged 9 to 26 years old, making the first option of its kind available for the country's male population.The vaccine was previously approved for use in females who are 9 to 45 years of age and has helped protect over 50 million women in China from certain HPV-related cancers and diseases since its first approval, Merck’s president of Human Health International, Joseph Romanelli, said in a release.Gardasil has recently seen slumping sales in China, where it's being marketed by Merck’s regional commercial partner Zhifei Biological Products. Rob David previously called the expansion to the male population a “significant opportunity.” UPDATED: Tuesday, Jan. 7 at 3:57 p.m. ETThe FDA tacked on additional warning information to the prescribing information for Agios’ pyruvate kinase (PK) deficiency drug Pyrukynd, flagging instances of liver deficiency in certain situations.The label update follows liver injury observations in patients who received Pyrukynd at a dose higher than is recommended for PK deficiency, according to an SEC filing from the company.The drug was approved by the FDA in 2022 as the first medicine to treat hemolytic anemia in adults with PK deficiency. Besides this use, the company is exploring expansions into beta thalassemia and sickle cell disease.The updated "Warnings and Precautions" section urges prescribers to run liver tests on patients before initiating treatment and monthly for the first six months of treatment. Patients should stop treatment if hepatic injury is suspected.Bayer is vying for a third indication for its Nubeqa (darolutamide) in China, this time for its use in combination with androgen deprivation therapy (ADT) in patients with metastatic hormone-sensitive prostate cancer.The company filed an application for the label expansion with China’s National Medical Products Administration, leveraging data from its phase 3 ARANOTE trial. In the study, Nubeqa and ADT significantly reduced the risk of radiological progression or death by 46% compared with placebo plus ADT.The nod would add to the drug’s two existing approvals in China, which cover other prostate cancer patient populations. It is estimated that the prevalence of prostate cancer in China will exceed 161,000 cases annually by 2026, according to Bayer. Dizal’s non-small cell lung cancer (NSCLC) candidate sunvozertinib will get a speedy FDA review after the agency granted priority review to the company’s new drug application.The company is specifically going after an approval to treat locally advanced or metastatic non-small cell lung cancer (NSCLC) in patients with epidermal growth factor receptor (EGFR) exon 20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy. Sunvozertinib won its world-first approval in China in 2023. UPDATED: Monday, Jan. 6 at 10:25 a.m. ETAmgen's DLL3-targeted bispecific antibody Imdylltra, known as Imdelltra in the U.S., has won a conditional marketing authorization from the U.K.'s Medicines and Healthcare products Regulatory Agency as a third-line therapy for small cell lung cancer.The conditional nod is based on results from the phase 2 DeLLphi-301 trial, which showed that the T-cell engager triggered a tumor response rate of 41%, with the median response lasting 9.7 months. It follows an FDA approval in May 2024.China has approved Astellas' first-in-class Claudin 18.2 drug Vyloy, following similar moves by drug regulators in Japan and the U.S.The drug's Chinese indication is in first-line, CLDN18.2-positive, HER2-negative advanced gastric or gastroesophageal junction adenocarcinoma. The CLDN18.2 biomarker is expressed by about 35% of Chinese stomach cancer patients, according to Astellas.The approval was backed by two global phase 3 trials, dubbed Glow and Spotlight, which evaluated Vyloy in combination with the chemotherapy regimens CAPOX and mFOLFOX6, respectively. Both trials linked the drug's chemo combo to longer life expectancy and progression-free survival time compared with chemo alone.UPDATED: Friday, Jan. 3 at 2:45 p.m. ETGSK’s IL-5 inhibitor Nucala scored a nod in China to treat people with inadequately controlled chronic rhinosinusitis with nasal polyps (CRSwNP), a condition that impacts nearly 30 million people in China.The drug was approved by China’s National Medical Products Administration for use as an add-on therapy along with intranasal corticosteroids for patients with CRSwNP who don’t achieve adequate disease control with systemic corticosteroids or surgery. The indication is Nucala’s third in China and comes after prior approvals in eosinophilic asthma and eosinophilic granulomatosis with polyangiitis.An FDA advisory committee panel is scheduled to discuss the merits of Novartis’ oral factor B inhibitor Fabhalta in the rare kidney disease C3 glomerulopathy (C3G).The Cardiovascular and Renal Drugs Advisory Committee will meet on February 24, according to an FDA document (PDF). The drug was approved by the agency in 2023 as a treatment for paroxysmal nocturnal hemoglobinuria, winning the title of the first oral monotherapy approved for the rare blood disorder. Fabhalta received a second nod for immunoglobulin A nephropathy (IgAN) in August.In studies, Fabhalta proved that it could reduce proteinuria for C3G patients, a marker for delaying progression to kidney failure. So far, there are no approved treatments for the ultra-rare disease, which causes about 50% of patients to progress to kidney failure within ten years of diagnosis. UPDATED: Thursday, Jan. 2 at 1:45 p.m. ETShortly before the new year, Merck & Co. scored an approval for its potential blockbuster Winrevair in the U.K. The pulmonary arterial hypertension (PAH) drug, which also goes by the name sotatercept, was originally picked up via Merck’s $11.5 billion acquisition of Acceleron back in 2021.The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) approved Winrevair in combination with other PAH therapies to improve exercise capacity in adult patients who live with moderate or marked limitations of physical activity.Patients with PAH suffer from high blood pressure in the blood vessels that supply the lungs, which can ultimately damage the right side of the heart. Winrevair, for its part, targets the causes of PAH responsible for the narrowing of the arteries in the lungs, in turn making it easier for the heart to pump blood to the pair of organs, according to the U.K. government’s release.The MHRA based its approval on a study of 323 PAH patients that saw Winrevair best placebo at improving exercise ability. The main measure of effectiveness in the trial was the difference in distance patients could walk in six minutes before and after treatment.At the trial’s 24-week treatment mark, patients on Winrevair plus other PAH medicines improved their walking distance by around 34 meters, versus one meter in patients who received placebo.Merck’s U.K. nod for Winrevair follows green lights from the U.S. FDA last March and the European Commission in August.At the time of the drug’s U.S. approval last year, J.P. Morgan’s Chris Schott estimated Winrevair could eventually reel in peak sales between $3 billion and $4 billion. 

核药领域正在进入收获期。 全球核药龙头捷报频传——诺华的两款核药2024年销售收入首次突破20亿美元；远大医药（0512.HK）交出的2024年答卷同样让人眼前一亮。 3月12日，远大医药发布2024年年度业绩，公司实现收入约116.45亿港元，剔除汇率同比增长12.8%；归母净利润约24.68亿港元，剔除汇率同比增长34%；得益于去年良好的业绩表现，公司拟派息每股0.26港元，合计超9.1亿港元。 这份成绩单发布后的首个交易日就获得了市场资金的强烈认可，远大医药股价一度大涨超过21%，最终当日收涨19.66%。 远大医药作为全球仅有的四家在肿瘤治疗方面成功实现创新核药商业化应用的创新制药企业之一，如今以易甘泰®钇[90Y]为首的核药板块已经成为公司收入增长最快的“尖兵”之一。同时，公司的全球化程度和核药管线数量在国际领先，目前拥有3款产品处在临床三期阶段，且核药产品储备数量已超过20个，未来像易甘泰®钇[90Y]这样的爆品，远大医药有望陆续推出。 立足当下，2025年将是远大医药业绩爆发的元年，公司凭借着“自研+合作引进+并购布局”多轮驱动投入转型创新的模式，2024年已经实现63项重大的里程碑，在年内新增14款商业化产品的同时，我们同时也看到公司的核药、五官科、呼吸等板块中的数款创新产品正在快步迈入商业化放量的新阶段，不仅将带动公司的收入规模快速增长，并且将公司整体的盈利结构和质量进一步优化提升。 远大医药即将迎来属于自己的股价和业绩的“戴维斯双击”时刻。 01 核药重磅炸弹时代来临 核药的重磅炸弹时代来临，远大医药将率先并充分受益。 2024年诺华两款核药收入达到21.16亿美元，其中Pluvicto实现销售13.92亿美元（同比增长42%），首次进入“10亿美元分子俱乐部”，而Lutathera得益于适应症范围的扩大（纳入了12岁及以上SSTR+胃肠胰神经内分泌肿瘤儿童患者），实现7.24亿美元（同比增长20%），在2-3年内也将进入10亿美元分子行列。 基于两款核药Pluvicto和Lutathera各自在前列腺癌、胃肠胰神经内分泌瘤领域展现出的强大治疗潜力和广阔的市场空间，近年来MNC和国际资本大力布局核药资产以“占位”下一个重磅炸弹，仅2024年MNC便完成了3笔核药并购交易，包括阿斯利康20亿美元收购Fusion Pharmaceuticals、BMS以41亿美元收购RayzeBio、诺华以10亿美元预付款+7.5亿美元里程碑收购Mariana Oncology。 远大医药在国内的强劲商业化表现，也正在兑现核药在全球市场所展现出的潜力。 2024年，远大医药核药板块实现收入约5.89亿港元，剔除汇率同比大幅增长近176.6%，其中易甘泰®钇[90Y]微球注射液自2022年国内获批上市后快速放量，已累计治疗近2000例患者，目前其正式手术已在国内22个省市的50余家医院展开。 易甘泰®钇[90Y]的快速放量，不仅基于远大医药出色的商业化，并且离不开核药自身治疗机制优势和优异的疗效解决临床未满足需求。 诺华的Pluvicto填补了PSMA阳性转移性去势抵抗性前列腺癌（mCRPC）治疗的临床空白，并在上市之初一度因产能问题导致供不应求。易甘泰®钇[90Y]在原发性肝癌和结肠癌肝转移领域为大量用药选择有限的患者提供了全新治疗选择，能够实现肝癌降期并帮助患者实现临床治愈，这是过去传统的治疗手段无法做到的。 易甘泰®钇[90Y]国内商业化以来已累计治疗近2000例患者，目前已有40余名患者顺利实现肝癌肿瘤降期转化并实施了肝癌切除手术，10余名患者通过桥接治疗成功实施肝移植，实现临床治愈。 易甘泰®钇[90Y]之外，随着远大医药与合作伙伴的快速临床推进，公司处于后期临床产品线正持续丰富，截至目前公司有3款核素偶联药物（RDC）处于临床三期，分别是前列腺癌诊断核药TLX591-CDx、透明细胞肾细胞癌诊断核药TLX250-CDx和胃肠胰神经内分泌瘤治疗性核药ITM-11，这也使得远大医药成为国内拥有最多三期临床创新核药管线的公司之一，足见公司在核药领域的深耕程度和布局的前瞻性。 这三款核药管线中TLX591-CDx、TLX250-CDx两款管线源自于与Telix的合作，而Telix在海外的表现已经充分证明了市场对其的认可和潜在巨大产品价值。 2024年，Telix总收入实现7.83亿澳元（约合4.87亿美元），同比增长56%；净利润约4990万澳元（约合0.31亿美元），同比增长860%。Telix业绩的高速增长的源于Illuccix®（即TLX591-CDx ）的持续放量，自2022年Illuccix®获批以来，其在美国展现出强劲的放量表现。这种强劲的放量表现，不仅仅是得益于Pluvicto的快速爆量，更在于产品的高灵敏度和特异性，其不仅能够检测到90%以上原发性和转移性前列腺癌细胞表面的PSMA阳性病变，可用于检测基于血清PSA水平升高的复发前列腺癌患者，这促使Illuccix®在美国诊断性核药的竞争中更获得医生青睐。 自Illuccix®商业化放量以来，Telix的股价累计涨幅已经超过了500%。Telix管理层在最新的交流中也给出了2025年的业绩指引：公司收入将达到11.8~12.3亿澳元（同比增长50.6~57.05%），并计划继续增加研发投入，预计同比增长20~25%。 除此之外，Telix准备在2025年推出三款新产品 TLX007-CDx (Gozellix®)、TLX101-CDx(Pixclara®) 和 TLX250-CDx (Zircaix®)，这对于远大医药来说显然是好消息，随着合作产品在美国实现商业化，国内管线可以通过桥接临床实现进度加速。 值得一提的是TLX250-CDx，目前透明细胞肾细胞癌（ccRCC）临床常用诊断方法CT或MRI提示存在肾脏肿块但无法判断是否为ccRCC的患者，TLX250-CDx正电子发射断层成像(PET)在诊断ccRCC的敏感性和特异性上分别达到86%和87%，远超过FDA要求的预设阈值，其有望成为ccRCC全新临床诊断标准。另外，TLX250-CDx并不局限于ccRCC，目前用于三阴性乳腺癌（TNBC）、非肌层浸润性膀胱癌（NMIBC）以及尿路上皮癌（UC）的多项适应症临床研究也在全球同步推进中。 在全球热门SSTR靶向核药布局方向，远大医药选择重点推进的治疗性核药ITM-11是一款潜在的同类最佳产品，这是一款将无载体177Lu与生长抑素类似物结合的RDC，全球三期临床COMPOSE研究显示，在侵袭性2/3级SSTR阳性的胃肠或胰腺来源的神经内分泌肿瘤GEP-NETs患者中，患者的mPFS高达34.5个月，且未观察到严重的不良事件。与Lutathera非头对头数据比较下，ITM-11有望在整体的疗效（mPFS显著提升，Lutathera一线治疗在22个月左右）、安全性上成为同类最佳产品。 远大医药不仅核药商业化拔得头筹，并且其核药诊疗一体化大平台已经逐渐成型，实现了自研&合作技术平台（20款储备管线）、温江研发生产基地、商业化平台的布局兑现，核药板块后劲很足。 02 新爆点：创新眼药攻势如潮 经历了数年的深耕与布局，2024年可谓是远大医药创新眼药的蓄力之年，未来几年眼科创新药物显然将成为公司的全新爆点。 过去，远大医药的五官科板块已经为公司在眼科商业化层面打下了坚实基础，已商业化的品种包括：国家保护独家品种的和血明目片（治疗眼底疾病）、治疗干眼症一线用药的人工泪液瑞珠等，这些均为畅销的大品种。 公司在多年的合作与沉淀后，已经构建起了一个丰富而具有差异化的创新眼药管线平台，从覆盖的疾病广度包括像屈光不正、干眼症这样的眼科大适应症，从差异化和未满足临床需求又可以覆盖到像蠕形螨睑缘炎、翼状胬肉这样的缺乏明确对症治疗药物的领域。 2024年至今，远大医药在创新眼药领域发生了许多重磅的里程碑事件，包括白内障术后抗炎镇痛创新药GPN00833完成国内三期临床研究并达到临床终点、从箕星药业引入OC-01和OC-02鼻喷雾剂两款干眼症药物的大中华区权益，以及蠕形螨睑缘炎创新眼药GPN01768的NDA获得CDE受理等，这几款创新药物未来的商业化放量都将给远大医药的眼药板块甚至公司整体业绩带来巨大的弹性。 博研咨询数据显示，2023年国内治疗药物市场高达120亿元，同比增长15%。其中，人工泪液在国内干眼症治疗市场中的占比超过50%。值得注意的是，当前市面上的人工泪液仅可模拟泪膜的一种或多种成分，难以真正对天然泪液进行替代。 远大医药已经商业化的OC-01（酒石酸伐尼克兰）是一种不含防腐剂鼻喷雾剂，其不同于人工泪液的眼表给药方式而采用创新的经鼻给药方式，其有效成份伐尼克兰通过结合烟碱型乙酰胆碱 (nACh) 使其活化，三叉神经副交感神经通路（控制泪膜自稳态）被激活，导致基础泪液分泌增加，从而达到治疗干眼的目的。 相比长期使用人工泪液可能产生的药物依赖、引发结膜炎和眼角膜损伤等安全性风险，OC-01拥有更高的选择性和安全性，其市场潜力非常可观。鉴于玻璃酸钠滴眼液、环孢素滴眼液在国内2023年销售规模均超过10亿元，OC-01凭借创新药属性和全新作用机制有望快速突破这一门槛。 远大医药前不久在三期临床取得成功的GPN00833是一款抗炎镇痛类激素纳米混悬滴眼液，目前激素眼用制剂是白内障手术后抗炎镇痛最常用且最有效的药物之一，可以快速有效控制眼科术后的感染、减轻患者眼部炎症和促进伤口愈合， 现有眼科术后围手术期激素眼用制剂（多为糖皮质激素，如地塞米松、泼尼松龙）普遍存在安全性风险较高的问题。 GPN00833利用独特的纳米制剂工艺解决了激素类产品低水溶性导致的生物利用度低及安全性风险，并在国内两项三期研究中其展现出可持久地清除眼部炎症和治愈眼部疼痛的疗效，同时安全性与安慰剂相似，有望对现有激素眼用制剂进行迭代。 博研咨询数据显示，2023年国内泼尼松龙滴眼液（专利期已过、单价35元/支）在8亿元左右。GPN00833作为安全性、有效性更优的迭代创新药，市场竞争格局更优、具备创新溢价，未来销售峰值超越泼尼松龙滴眼液只是时间问题。 处于NDA审评阶段的GPN01768（Xdemvy）是一种非竞争性γ-氨基丁酸氯离子通道（GABA-Cl）拮抗剂，用于治疗蠕形螨睑缘炎。蠕形螨睑缘炎是一种容易复发的疾病，影响的人群数量庞大，在中国和美国影响人群分别为4000万人和2500万人，在GPN01768获批之前全球尚未有针对蠕形螨睑缘炎的药物上市，临床空白巨大。 GPN01768在中国和美国的临床充分证明了自身对症杀螨的强大有效性和安全性，尤其国内三期临床研究LIBRA的顶线数据结果显示，GPN01768治疗蠕形螨睑缘炎患者的螨虫根除率具有统计学显著性（p<0.001），关键临床的大多数不良事件轻微可控，未出现治疗相关的停药。 GPN01768早在2023年7月在美国实现商业化，上市以来快速放量，2024年实现销售1.8亿美元，美银美林BofA更是预测GPN01768的峰值销售额将高达11亿美元。基于该药在美国上市以来的快速放量趋势、国内患者人群基数大于美国，其在国内的销售峰值潜力显然在10亿元之上。 集合上述分析，远大医药这三款商业化阶段创新眼药产品再加上治疗翼状胬肉的GPN00153（CBT-001）合计有着超越50亿国内销售的潜力，而这些差异化品种的合作引进，仅仅也只是公司前瞻性布局眼光和差异化品种战略的冰山一角的早期兑现，随着这些品种的商业化放量能力逐步验证以及公司自研产品的发力，远大医药的创新眼药板块成长性将会越来越强。 03 两大板块核心优势继续强化 远大医药布局特点是较快的并购节奏和明确指向补强，重点在于提升呼吸及危重症板块丰度，以及强化公司在心脑血管急救板块的竞争力。 呼吸、心脑血管这两大领域均为致死风险极高的疾病领域，临床未满足需求多，药物开发市场空间大。 2024年远大医药呼吸及危重症板块的表现突出，实现营收17.09亿港元，同比增长26.9%，展现了十足的成长力。这种稳定的成长力来源于公司深厚的积累，目前公司手握切诺®（黏液溶解促排剂）、恩卓润®和恩明润®（改善中重度哮喘）等稳定贡献现金流的品种，还有诸如Ryaltris®这样的创新品种正在蓄势待发。 在该领域，远大医药2024年完成了对百济制药的全资并购，在获得百济制药技术领先鼻喷制剂平台的同时，通过纳入该公司管线资产，形成了一套面向轻、中、重度过敏性鼻炎患者的吸入制剂药物产品组合。 远大医药治疗过敏性鼻炎鼻喷剂Ryaltris®正等待CDE的上市批准，作为国内第一个创新复方鼻喷剂其填补了一线对症治疗经治中重度过敏性鼻炎患者的临床空白。公司通过并购百济制药获得了一线过敏性鼻炎鼻喷剂用药（丙酸氟替卡松鼻喷雾剂、糠酸莫米松鼻喷雾剂、布地奈德鼻喷雾剂），这一产品组合未来要分享的是国内过敏性鼻炎百亿的用药市场，并购带来的化学反应不可估量。 另外，远大医药针对未满足临床需求如重症治疗监护、呼吸窘迫综合征、脓毒症等领域开发了拥有自主知识产权的“全球新”药物，如脓毒症创新药STC3141已完成国内二期临床患者入组并给药、脓毒症创新药APAD已完成了国内一期临床并顺利达成临床终点。 在心脑血管急救板块，远大医药近年来品种数量正在快速扩充（心脑血管急救板块从2022H1的24个品种到2024年的30个品种），产品管线数量位居行业前列，覆盖血压控制、血管活性药、心肌代谢、心衰、抗凝等领域，后劲充足。 2024年，远大医药该板块力美通®依普利酮片成功进入国家医保目录，进一步促进了产品可及性的提升。同时，公司通过布局天津田边合心爽®、合贝爽®这样的畅销心脑血管急救药物（临床上治疗冠状动脉痉挛心绞痛的首选药物）迅速切入如如糖尿病、胃肠道疾病等慢性疾病赛道，同时获得天津天边的制剂产能加速公司原料制剂一体化，提升公司的盈利能力。另外，远大医药同年收购了多普泰，其核心产品脉血康系列药物（销售规模在5亿元）及其他产品与公司现有产品组合也形成了良好的协同效应。 随着远大医药的创新急救产品Jext®在大湾区获批上市，叠加这两大并购资产的注入，未来几年将成为公司全新增长动力，带来一揽子有充分竞争力的产品，为板块业绩成长提供坚实保障。 从对远大医药的呼吸及危重症、心脑血管急救这两大优势板块布局分析不难看出，公司在过去的投入及积累了大量的现金牛产品，已经逐渐放量成为各板块业绩的稳定内生增长动力；而公司通过自研、引入具备差异化的创新产品，同时辅以补强指向明确的资产并购，使得公司在呼吸、心脑血管这两大板块未来不缺乏“强大的发动机”，这显然能够双轮驱动公司板块以及整体业绩快速提升。 结语：历经多年的投入、前瞻性的布局眼光和强大商业化并购整合能力，远大医药的核药、眼药、呼吸和心脑血管等几大板块正在快速步入创新布局兑现期。核药板块易甘泰®钇[90Y]有望进一步放量冲击10亿单品，成都温江甲级核素研发生产基地将在2025年建成形成集研发、生产、运营为一体的全产业链版图；呼吸及危重症板块，STC3141国内脓毒症二期有望在上半年取得初步成药性数据；五官科板块，新一代干眼症药物商业化加速放量，多款创新眼药将迎来重大里程碑；另外，心脑血管精准介入、生物科技板块，公司也在丰富产品技术布局和积极拓展商业化链路，为公司增长贡献稳健增量。 可以预见的是，远大医药2024年交出的这一份出色的业绩答卷也仅仅是这艘巨轮加速行驶的预兆，而更多的惊喜和爆发式成长或许还在后头，我们或许能见证一家传统Pharma转型创新成功的涅槃过程。 识别微信二维码，添加抗体圈小编，符合条件者即可加入抗体圈微信群！ 请注明：姓名+研究方向！ 本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。