Ceftobiprole Medocaril Sodium

2024’s novel drug approvals offered a wide array of new cancer drugs, rare disease treatments, a potential Alzheimer’s blockbuster, and about 10 fewer approvals than 2023. While 2022 and 2023 saw major approvals of new cell and gene therapies, 2024 did not stick as cleanly to a theme — instead featuring a grab-bag of long-awaited approvals. That included Eli Lilly’s amyloid-targeted Alzheimer’s drug, the first two treatments for the rare Niemann-Pick disease, and the first-ever mesenchymal stromal cell therapy. Several approvals in 2024 also deployed new ways to treat familiar diseases. For instance, Pfizer’s new hemophilia A or B drug Hympavzi is the first to target a particular protein in the blood-clotting process. Bristol Myers Squibb’s Cobenfy, approved for schizophrenia, is the first antipsychotic that targets cholinergic receptors and not dopamine receptors. What to watch for in 2025: more cancer drug approvals and major label expansions. And answers to questions like: Will the incoming Trump administration push to overturn some drug and vaccine approvals or EUAs? Or attempt to make the approval process more difficult? — Zachary Brennan Active ingredient: berdazimer sodium Indication: Molluscum contagiosum in adults and pediatric patients 1 year of age and older Snapshot : Ligand Pharmaceuticals scored FDA approval in January for berdazimer sodium, marketed as Zelsuvmi, to treat the viral skin infection molluscum contagiosum. The topical gel was approved for at-home treatment in both adult and pediatric patients ages 1 one year and older and is the first novel drug for these types of infections. The approval came after Ligand acquired the assets of Novan Pharmaceuticals, which had initially overseen the drug’s development and filed for bankruptcy in July 2023. — Lia DeGroot Active ingredient: lifileucel Indication: Adult patients with unresectable or metastatic melanoma who have been previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor. Snapshot : An idea more than 40 years in the making finally went commercial in February with the approval of the first treatment based on tumor-infiltrating lymphocytes. Iovance’s bespoke therapy, Amtagvi, is made by dissecting out immune cells from a patient’s tumor, bolstering their numbers in a lab, and reinfusing the cells into a patient to help keep cancer at bay. The company is testing the approach in other cancers and as a frontline therapy for melanoma. The Biosecure Act threatened Iovance’s relations with the Chinese contract manufacturer WuXi, which helped make Amtagvi, but sales of the therapy were still off to a strong start with $54.9 million in total revenue by the end of the third quarter. — Ryan Cross Active ingredient: cefepime and enmetazobactam Indication: Complicated urinary tract infections in patients 18 years and older Snapshot : Exblifep, a combination of cefepime and enmetazobactam delivered intravenously, was approved in February for adults with complicated urinary tract infections, including pyelonephritis, or kidney infection. Enmetazobactam was discovered by Orchid Pharma, which licensed the ex-India rights to Allecra in 2013. — Nicole DeFeudis Active ingredient: letibotulinumtoxinA-wlbg Indication: To treat moderate-to-severe frown lines in adults Snapshot : Hugel Aesthetics’ Botox competitor Letybo was approved in March for the treatment of moderate to severe frown lines. Letybo was initially rejected in 2022 over manufacturing issues. The FDA turned it down again the following year, and Hugel refiled it in September 2023 after “completing a review of the facility management and supplementary work.” Despite a growing number of Botox rivals on the market, AbbVie’s chief commercial officer Jeffrey Stewart said at the 2024 JP Morgan Healthcare Conference that the company has been “incredibly efficient” at maintaining Botox’s lead. — Nicole DeFeudis Active ingredient: resmetirom Indication: Noncirrhotic non-alcoholic steatohepatitis (NASH) with moderate-to-advanced liver scarring in adults Snapshot : Madrigal Pharmaceuticals’ resmetirom won accelerated approval in March as the first treatment for metabolic dysfunction-associated steatohepatitis (MASH), the debilitating liver disease also called NASH. The approval is likely to be seen as a crucial milestone in MASH, where the standard of care was previously diet and exercise. Rezdiffra, as the drug is marketed, is indicated for patients with stage 2 or 3 fibrosis who have not progressed to cirrhosis. — Nicole DeFeudis Active ingredient: tislelizumab Indication: Unresectable or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic chemotherapy that did not include a PD-(L)1 inhibitor Snapshot : BeiGene had licensed US rights to its PD-1 inhibitor twice during the development journey, once to Celgene and the other time to Novartis. By the time the biotech clinched FDA approval for Tevimbra, it became the sole owner again. While Novartis and BeiGene had first filed for approval more than two years prior, the FDA was unable to inspect BeiGene’s manufacturing site in China amid the pandemic, leading to a lengthy review delay. As with the many checkpoint drugs already on the market, BeiGene hopes esophageal cancer will be the first of many indications Tevimbra will eventually be approved to treat, both on its own and in combination with other medicines. The drug was first approved in China in 2019. — Amber Tong Active ingredient: atidarsagene autotemcel Indication: Children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy Snapshot : With a $4.25 million price tag, Lenmeldy is on the list of the world’s most expensive medicines. The gene therapy is for an ultra-rare and fatal disease called early-onset metachromatic leukodystrophy, which affects fewer than 40 children a year in the US. Lenmeldy was already approved under a different brand name in the EU and UK and was shown in clinical trials to keep the majority of children who received it alive and with normal cognitive development. Japanese pharmaceutical company Kyowa Kirin completed its purchase of the company that developed the therapy, Orchard Therapeutics, last January after Orchard had been struggling to build a sustainable business around commercializing these complex treatments for rare diseases with small numbers of patients. — Lei Lei Wu Active ingredient: aprocitentan Indication: Hypertension in combination with other antihypertensive drugs, to lower blood pressure in adult patients who are not adequately controlled on other drugs Snapshot : The FDA approved Idorsia’s Tryvio for patients who aren’t adequately treated by current blood pressure medications. Johnson & Johnson backed away from its partnership on the drug in 2022, and while the pharma will still receive royalties, Idorsia needs a new partner to sustain its operations through this year. Idorsia said in November that it entered exclusive negotiations with an undisclosed third party for global rights to the drug, netting $35 million, and that it could lay off approximately 270 staffers. The drug carries a black box warning for embryo-fetal toxicity and is only available via a restricted safety program. The FDA only approved the lower dose that Idorsia studied in its key clinical trial. — Lei Lei Wu Active ingredient: givinostat Indication: Duchenne muscular dystrophy in patients 6 years of age and older. Snapshot : The FDA’s approval of Duvyzat marked the first commercial nonsteroidal drug for all genetic variants of Duchenne muscular dystrophy. The treatment, an oral histone deacetylase inhibitor developed by Italfarmaco, was approved for Duchenne patients aged 6 and older. Unlike genetic medicines for Duchenne, such as exon-skipping drugs or gene therapy, Duvyzat aims to counteract the downstream effects of missing dystrophin, a crucial muscle protein, rather than trying to restore it. In clinical trials, Duvyzat led to improved performance on a stair climb test that’s used to measure motor function in patients with muscle diseases. — Lei Lei Wu Active ingredient: sotatercept Indication: Pulmonary arterial hypertension in adults Snapshot : Merck’s $11.5 billion Acceleron deal appeared to pay off with the much-anticipated approval of its pulmonary arterial hypertension drug Winrevair. The rare disease medicine, which had a “successful resurrection” around mid-stage data in 2020, is expected to become a quick blockbuster treatment as the New Jersey pharma looks to fill the eventual patent gap with Keytruda. The under-the-skin drug, when combined with background PAH therapy, helped patients walk farther than patients on placebo and background treatment in a Phase 3 trial. “The increase in six-minute walk distance capability was gargantuan,” Eliav Barr, chief medical officer at Merck Research Laboratories, said in an interview with Endpoints News . — Kyle LaHucik Active ingredient: vadadustat Indication: Anemia from chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months Snapshot : After securing approval in more than 35 other countries, Akebia’s anemia drug finally got the FDA’s blessing in March, almost two years to the day after it was initially rejected by the agency. The once-daily Vafseo tablets were greenlit for adults who have been on dialysis for at least three months and indicated for anemia due to chronic kidney disease. The drug belongs to a class of medicines known as hypoxia-inducible factor prolyl hydroxylase inhibitors. They’ve had a precarious history. In 2021, FibroGen’s drug was also rejected by the FDA. GSK, meanwhile, won US approval in February 2023, but wound up ditching plans to seek European approval. Akebia doesn’t plan on making the drug available in the US until this month. — Kyle LaHucik Active ingredient: danicopan Indication: Add-on therapy for extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH) Snapshot : The oral factor D inhibitor Voydeya from AstraZeneca scored approval in the US in April 2024 as an add-on therapy to Soliris and Ultomiris for the treatment of a rare blood disorder called extravascular hemolysis. Voydeya is also being developed as a monotherapy for an advanced form of age-related macular degeneration. — Katherine Lewin Active ingredient: ceftobiprole Indication: Adults with Staphylococcus aureus bloodstream infections; adults with acute bacterial skin and skin structure infections; and adult and pediatric patients 3 months to less than 18 years old with community-acquired bacterial pneumonia Snapshot : The FDA approved Zevtera for staph infections, acute bacterial skin and skin structure infections, and community-acquired bacterial pneumonia. The approval came later than planned — Basilea initially said that it would submit the drug to the FDA in early 2023, but pushed back those plans after manufacturing issues. — Katherine Lewin Active ingredient: nogapendekin alfa inbakicept-pmln Indication: Adult patients with BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumors Snapshot : Anktiva was approved in combination with Bacillus Calmette-Guérin (BCG) to treat patients with a subtype of BCG-unresponsive non-muscle invasive bladder cancer. ImmunityBio was hit with an FDA rejection in May 2023, citing manufacturing issues with a third party. After the eventual approval, ImmunityBio received $100 million as part of a royalty deal and private placement with Oberland Capital. — Katherine Lewin Active ingredient: tovorafenib Indication: Children 6 months of age and older with low-grade glioma that has stopped responding to or returned after previous treatment Snapshot : Day One Biopharmaceuticals’ goal is to develop new drugs for children first — reversing the industry’s age-old practice of first winning approval of medicines in adults and then (and only on occasion) in children. The company’s first approved drug, Ojemda, is a targeted treatment called a BRAF inhibitor. It was approved for children 6 months of age and up with a form of slow-growing brain cancer called low-grade glioma. “We now have shown that you can develop drugs intentionally for children at the speed of biotech,” Day One’s then-R&D chief Sam Blackman told Endpoints around Ojemda’s approval. — Lei Lei Wu Active ingredient: pivmecillinam Indication: Uncomplicated urinary tract infections caused by susceptible isolates of Escherichia coli , Proteus mirabilis and Staphylococcus saprophyticus , in female patients 18 years and older Snapshot : In April, Utility Therapeutics’ Pivya became the first new treatment for uncomplicated urinary tract infections in more than two decades. The oral antibiotic induced a composite response in 62% of patients versus just 10% for placebo in one of Utility’s three registrational trials. The UK-based drugmaker bought the US rights to pivmecillinam — the active ingredient in Pivya — from Leo Pharma as part of a 2018 deal with undisclosed financial terms. — Ayisha Sharma Active ingredient: fidanacogene elaparvovec-dzkt Indication: Moderate-to-severe hemophilia B in adults Snapshot : Pfizer became the second company to nab FDA approval for a one-time gene therapy to treat hemophilia B. And just like the US market’s first entrant, CSL, Pfizer priced the treatment at an eye-popping $3.5 million. Pfizer’s Beqvez was found to eliminate bleeding in 60% of patients in an open-label Phase 3 study, compared to 29% of patients receiving prophylaxis with a factor IX inhibitor. While it won approval for Beqvez, Pfizer has taken a step back from the gene therapy field, selling early assets in 2023 and dropping its Duchenne gene therapy after a failed trial. — Lei Lei Wu Active ingredient: mavorixafor Indication: WHIM syndrome in patients 12 and older Snapshot : Xolremdi was approved for an ultra-rare immune disorder, WHIM, named for its symptoms: warts, hypogammaglobulinemia, infections, and myelokathexis. X4 Pharmaceuticals’ drug is a CXCR4 inhibitor that is meant to address the underlying cause of the disease and improve patients’ ability to fight infections. X4 is also exploring Xolremdi’s use in treating chronic neutropenia. — Lei Lei Wu Active ingredient: tarlatamab-dlle Indication: Extensive-stage small cell lung cancer with disease progression on or after platinum-based chemotherapy. Snapshot : Imdelltra’s approval marked a major milestone for bispecific T cell engagers, a class of antibody-based drugs that bring T cells to tumors in hopes that they will attack the cancer. Following Immunocore’s Kimmtrak, it became the second T cell engager approved to treat solid tumors. Imdelltra was approved for extensive-stage small cell lung cancer that progressed despite chemotherapy, and Wall Street analysts project that the therapy has blockbuster potential if it can move into earlier lines of treatment. — Lei Lei Wu Active ingredient: RSV vaccine Indication: Prevention of lower respiratory tract disease caused by respiratory syncytial virus (RSV) in individuals 60 and older Snapshot : Moderna expects its RSV vaccine, its first non-Covid approval, to play a significant role in the company’s return-to-growth strategy by 2026 and reverse the $4.7 billion loss it recorded in 2023. The first full RSV vaccine season (winter 2023-24) was highly successful for Moderna’s competitors, GSK and Pfizer, and analysts anticipate the market will grow to $10 billion over the next few years. — Max Gelman Active ingredient: imetelstat Indication: Adults with low- to intermediate- risk myelodysplastic syndromes with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents Snapshot : Geron’s imetelstat cleared an adcomm and then secured an FDA nod in June for certain anemic myelodysplastic syndrome patients who are dependent on blood transfusions. Branded as Rytelo, the telomerase inhibitor carries a $2,471 price tag for a 47 mg vial and costs $9,884 for a 188 mg vial. The approval came after a panel of experts voted 12 to 2 in March that the drug’s benefits outweigh its risks. — Ayisha Sharma Active ingredient: elafibranor Indication: Primary biliary cholangitis in combination with ursodeoxycholic acid in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA Snapshot : Ipsen’s $136 million bet on Genfit’s elafibranor paid off in June when the drug clinched US approval for primary biliary cholangitis, a rare disease that can lead to liver failure. The drug, sold as Iqirvo, is a first-in-class dual agonist of the alpha and delta isoforms of PPAR and could slow disease progression, according to Ipsen. PBC affects around 100,000 people in the US. — Ayisha Sharma Active ingredient: immune globulin intravenous, human-dira Indication: Primary humoral immunodeficiency (PI) in patients 2 or older Snapshot : Yimmugo was approved in June for patients 2 years and older with primary humoral immunodeficiency, a condition that affects the immune system and can leave patients at an increased risk of infections. Italy-based Kedrion Biopharma announced in July that it struck an agreement for the full commercialization and distribution of Yimmugo in the US. — Nicole DeFeudis Active ingredient: pneumococcal 21-valent conjugate vaccine Indication: For prevention of invasive pneumococcal disease and pneumococcal pneumonia. Snapshot : Merck’s 21-valent pneumococcal vaccine Capvaxive was specifically designed for adults. The strains covered by the vaccine were responsible for 84% of invasive pneumococcal disease in patients 50 years and older between 2018 and 2021, according to Merck. That includes 10 strains in common with Pfizer’s rival 20-valent shot Prevnar 20, plus 11 unique strains. — Nicole DeFeudis Active ingredient: sofpironium Indication: Primary axillary hyperhidrosis in adult and pediatric patients 9 years of age and older Snapshot : Botanix Pharmaceuticals unlocked a potential market of 10 million people in the US with the June approval of sofpironium for primary axillary hyperhidrosis, or excessive underarm sweat. Sold as a gel with the brand name Sofdra, Botanix launched an early patient experience program in the third quarter, followed by a broader launch in the fourth quarter, which is when it also expected its first revenues from the product. — Ayisha Sharma Active ingredient: crovalimab-akkz Indication: Adult and pediatric patients 13 years and older with paroxysmal nocturnal hemoglobinuria and body weight of at least 40 kg Snapshot : The FDA approval for Genentech’s C5 inhibitor crovalimab for people with paroxysmal nocturnal hemoglobinuria in June came with a significant caveat — the drug can only be given under a REMS pathway due to the serious risk of meningococcal infections. Two months after its US approval, crovalimab, sold as PiaSky, secured an EU green light for the rare disease. When left untreated, PNH causes intravascular hemolysis, thromboembolic events and cytopenia. — Ayisha Sharma Active ingredient: ensifentrine Indication: Maintenance treatment of chronic obstructive pulmonary disease in adult patients Snapshot : Verona Pharma launched its COPD drug Ohtuvayre in 2024, taking a step into a titan-filled arena. Known generically as ensifentrine, Ohtuvayre is a maintenance treatment for COPD, taken via nebulizer twice a day, that can be added to standard COPD therapies. Verona aims to carve out a niche for Ohtuvayre as an add-on treatment rather than competing directly with major pharma companies in COPD like GSK and AstraZeneca. — Lei Lei Wu Active ingredient: donanemab Indication: Alzheimer’s disease Snapshot : Eli Lilly has invested more than $8 billion over more than three decades in its search for an Alzheimer’s drug. It finally found success in July when the FDA approved Kisunla . Kisunla is the second amyloid-targeting antibody to get full FDA approval after Eisai and Biogen won approval for Leqembi last year. Experts once worried about Alzheimer’s drugs bankrupting Medicare, but sales of the drugs are off to a slower-than-expected start , partly due to the risk of potentially fatal brain bleeding and swelling — which occurred in about 1 in 300 people in Lilly’s study. Lilly hopes the drug will be safer and more effective when taken earlier, before symptoms of dementia begin. A large study testing that hypothesis is underway and is expected to wrap up in 2027. — Ryan Cross Active ingredient: deuruxolitinib Indication: Adults with severe alopecia areata Snapshot : Sun Pharma’s $576 million acquisition of Concert Pharmaceuticals in 2023 paid off with the approval of the JAK inhibitor Leqselvi in severe alopecia areata. Sun’s drug could compete against some big players, including Eli Lilly’s JAK inhibitor Olumiant and Pfizer’s Litfulo. However, the drug’s launch was delayed after Incyte, which makes a similar drug, secured an injunction against its sale in November. Sun said it planned to appeal. — Katherine Lewin Active ingredient: benzgalantamine Indication: Mild to moderate dementia of the Alzheimer’s type in adults Snapshot : Zunveyl is a new twist on an old pill that’s commonly used to help reduce some symptoms of Alzheimer’s disease, but potentially with fewer gastrointestinal side effects. The compound is a prodrug of galantamine, an acetylcholinesterase inhibitor approved in 2001 that blocks breakdown of a key neurochemical that brain cells use to communicate with each other. Alpha Cognition, a tiny Canadian company that won approval for Zunveyl in July based on bioavailability studies, said it will begin selling Zunveyl in early 2025. — Ryan Cross Active ingredient: afamitresgene autoleucel Indication: Unresectable or metastatic synovial sarcoma in adults have received prior chemotherapy, are HLA antigen(s) A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive, and whose tumor expresses the MAGE-A4 antigen as determined by FDA authorized companion diagnostic devices. Snapshot : Tecelra became the first cell therapy based on T cell receptors and the second cell therapy for a solid tumor cancer to win approval in the US in 2024. The therapy, known as afami-cel, is meant for a subsection of synovial sarcoma patients whose cancer has spread throughout their body or can’t be removed via surgery, and who have already received chemotherapy. Patients need to be tested for expression of a protein called MAGE-A4, which is found in a range of tumors, as well as an immune marker called HLA-A*02. — Lei Lei Wu Active ingredient: vorasidenib Indication: For adult and pediatric patients 12 years and older with grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, following surgery including biopsy, sub-total resection, or gross total resection. Snapshot : Brain cancer is getting its targeted therapies, too. Servier’s Voranigo was approved for people with slow-growing gliomas that bear an IDH mutation. Patients are eligible for the drug after surgery for their brain tumor. Over 80% of grade 2 gliomas bear an IDH mutation, and the FDA said this was the first approved systemic drug for this subset of patients. In a clinical trial versus placebo, the treatment staved off cancer progression for a median 16.6 months longer, which physicians called a “striking” result. — Lei Lei Wu Active ingredient: denileukin diftitox-cxdl Indication: Adult patients with relapsed or refractory stage I-III cutaneous T cell lymphoma after at least one prior systemic therapy Snapshot : Following a delay from a CRL over manufacturing issues in 2023, Lymphir gained FDA approval in August. In order to support the launch of the treatment for a rare cancer, the company’s cancer subsidiary, Citius Oncology, combined with TenX Keane Acquisition. — Katherine Lewin Active ingredient: palopegteriparatide Indication: Hypoparathyroidism in adults Snapshot : Ascendis rebounded from an earlier FDA rejection and subsequent delay of its new application to snag FDA approval of hypoparathyroidism drug Yorvipath. It was already approved in Austria and Germany, generating €1.5 million ($1.6 million) in the first quarter. Yorvipath’s US entry replaces a void left by Takeda’s Natpara, some two years after it was pulled off the market. Ascendis said at the time of approval that it was completing manufacturing of the product and expecting the first batch of supply to be available in the first quarter of 2025. — Max Bayer Active ingredient: nemolizumab Indication: Prurigo nodularis in adults Snapshot : In August, Galderma secured first-in-class status with the US approval of its IL-31 antibody nemolizumab for prurigo nodularis. The green light is the first step in unlocking Nemluvio’s blockbuster potential, much of which lies in eczema, according to Galderma. The Swiss drugmaker earned a label expansion for moderate-to-severe atopic dermatitis in December, with Jefferies analysts projecting $2.1 billion in global peak sales for the drug. — Ayisha Sharma Active ingredient: seladelpar Indication: Primary biliary cholangitis in combination with ursodeoxycholic acid, in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA Snapshot : After acquiring seladelpar in the $4.3 billion purchase of CymaBay Therapeutics, Gilead snagged FDA accelerated approval to treat PBC, boosting its liver portfolio. The drug joins three others on the market, though its ability to treat itch symptoms gives Gilead hope that it can edge out competitors. That potential benefit has also contributed to Livdelzi’s price, which is slightly higher than the other two approved therapies. — Max Bayer Active ingredient: axatilimab-csfr Indication: Chronic graft-versus-host disease after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg Snapshot : Thanks to the help of Syndax Pharmaceuticals, Incyte was able to get another cGVHD treatment approval in August. It already markets Jakafi. The newly approved CSF-1 receptor-blocking antibody will also be studied alongside Jakafi and in combination with steroids. Incyte has said it plans to target the approximately 6,500 people who have failed two prior drugs. It also hopes to get the medicine greenlit for idiopathic pulmonary fibrosis. — Kyle LaHucik Active ingredient: lazertinib Indication: Locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor exon 19 deletions or exon 21 L858R substitution mutations, as detected by an FDA-approved test Snapshot : Lazcluze was approved in August in combination with Johnson & Johnson’s Rybrevant for certain non-small cell lung cancer patients with two common EGFR mutations. The approval puts the combination therapy in direct competition with AstraZeneca and its blockbuster Tagrisso, which has been approved in the same lung cancer population since 2018. Tagrisso also recently won a label expansion as part of a chemotherapy combination regimen in that indication. — Nicole DeFeudis Active ingredient: lebrikizumab Indication: Moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable, in adult and pediatric patients 12 years and older who weigh at least 40 kg Snapshot : Following an FDA rejection over manufacturing issues in 2023 (and no concerns with safety or labeling), Lilly scored approval for Ebglyss in September for moderate-to-severe eczema. Between two studies, 38% of patients on Lilly’s drug achieved clear or almost-clear skin at 16 weeks. — Katherine Lewin Active ingredient: arimoclomol Indication: Neurological manifestations of Niemann-Pick disease type C in adult and pediatric patients 2 and older, in combination with miglustat Snapshot : Zevra Therapeutics won approval for Miplyffa, known generically as arimoclomol, for people with the rare genetic disorder Niemann-Pick disease type C who are aged 2 years and older. Miplyffa’s approval came after the FDA previously rejected the drug in 2021, but Zevra returned to ask for approval with a reanalysis of clinical trial data that showed improvements in swallowing, which can be affected by the disease. FDA hosted an advisory committee ahead of its decision that voted in favor of the drug for the disease, a lysosomal storage disorder in which the body can’t properly recycle certain fatty substances. — Lei Lei Wu Active ingredient: levacetylleucine Indication: Neurological manifestations of Niemann-Pick disease type C in adults and pediatric patients weighing ≥15 kg Snapshot : In September, IntraBio’s levacetylleucine became the second drug to clinch FDA approval for Niemann-Pick disease type C in the span of just one week. Branded Aqneursa, the oral drug can be taken up to three times daily with weight-dependent dosing. — Ayisha Sharma Active ingredient: xanomeline and trospium chloride Indication: Schizophrenia in adults Snapshot : Cobenfy’s approval gives Bristol Myers a significant foothold in the neuroscience and psychiatry fields, as it brings to market the first new schizophrenia drug in decades. It also validates the company’s $14 billion acquisition of Karuna, with execs predicting peak sales in the multibillion-dollar range. Researchers hope to broaden the use of Cobenfy in at least half a dozen other indications. — Max Gelman Active ingredient: inavolisib Indication: For adults with endocrine-resistant, PIK3CA-mutated, hormone receptor-positive, human epidermal growth factor receptor 2-negative, locally advanced or metastatic breast cancer, as detected by an FDA-approved test, following recurrence on or after completing adjuvant endocrine therapy Snapshot : Roche’s Itovebi, which the company says is the first targeted therapy approved for people with HR-positive disease, got the green light from the FDA in October for patients with PIK3CA-mutated HR-positive HER2-negative breast cancer in combo with chemotherapy and Pfizer’s Ibrance. Patients on the drug saw progression-free survival of 15 months and a 58% overall response rate. — Katherine Lewin Active ingredient: marstacimab-hncq Indication: For routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years and older with: hemophilia A without factor VIII inhibitors, or hemophilia B without factor IX inhibitors Snapshot : Pfizer had a few successes in hemophilia in 2024, first with a gene therapy approval in April and then the nod for Hympavzi. The drug reduces a naturally occurring anticoagulation protein, unlike other treatments, which replace a clotting factor. But the results over routine prophylaxis were “modest,” TD Cowen analysts have said. Pfizer will likely have to rely on its packaging as a pre-filled, auto-injector pen to get traction in the market, the analysts said. — Kyle LaHucik Active ingredient: zolbetuximab Indication: In combination with fluoropyrimidine- and platinum-containing chemotherapy for the first-line treatment of adults with locally advanced unresectable or metastatic human epidermal growth factor receptor 2-negative gastric or gastroesophageal junction adenocarcinoma whose tumors are Claudin18.2-positive, as determined by an FDA-approved test Snapshot : Vyloy, which targets a protein called Claudin18.2, scored an approval in combination with chemotherapy for the first-line treatment of locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma. The drug was approved in Europe and Japan as well, making it the first in its class to get worldwide approval. — Katherine Lewin Active ingredient: sulopenem etzadroxil and probenecid Indication: Uncomplicated urinary tract infections caused by the designated microorganisms Escherichia coli , Klebsiella pneumoniae , or Proteus mirabilis in adult women who have limited or no alternative oral antibacterial treatment options Snapshot : Iterum’s oral sulopenem was approved in October for certain patients with uncomplicated urinary tract infections (uUTIs). The drug, to be marketed as Orlynvah, is indicated specifically for patients who have limited or no alternative oral options. While the company had applied for broad use in adults with uUTIs, Iterum CEO Corey Fishman said the FDA’s label restrictions are “very consistent with how we’ve been thinking the whole time.” The agency had previously raised concerns that “inappropriate and/or extensive use” of an oral penem could result in cross-resistance with carbapenems, a class of antibiotics used for severe infections. — Nicole DeFeudis Active ingredient: obecabtagene autoleucel Indication: Adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia Snapshot : Autolus is challenging one of Gilead’s CAR-T cell therapies, Tecartus, with its recently approved treatment Aucatzyl in adult B-ALL. Aucatzyl will be listed at $525,000, making it pricier than Tecartus, but Autolus is hoping that its therapy’s safety profile will win over payers and providers. — Lei Lei Wu Active ingredient: eladocagene exuparvovec-tneq Indication: Adult and pediatric patients with aromatic 13 L-amino acid decarboxylase deficiency Snapshot : Known as Upstaza in Europe and Israel, PTC Therapeutics’ rare disease gene therapy finally won approval in the US in November under the brand name Kebilidi. The gene therapy is for AADC deficiency, an ultra-rare disease that impacts the nerve cells and brain. In the US, the therapy’s approval included children and adults across “the full spectrum of disease severity,” whereas the treatment’s previous approvals outside the country included patients with severe AADC deficiency. — Lei Lei Wu Active ingredient: revumenib Indication: Relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene translocation in adult and pediatric patients 1 year and older Snapshot : Revuforj, also known as revumenib, is the first drug approved in a new class of medicines called menin inhibitors. It’s used for an aggressive form of leukemia driven by rearrangement of the KMT2A gene. However, it carries a boxed warning for differentiation syndrome, a potentially life-threatening reaction to some blood cancer treatments. Syndax plans to seek approval in 2025 for leukemia patients with a different and more common genetic alteration called NPM1. — Nicole DeFeudis Active ingredient: zanidatamab Indication: Adults with previously treated, unresectable or metastatic HER2-positive biliary tract cancer (BTC), as detected by an FDA-approved test Snapshot : Jazz Pharmaceuticals’ HER2-targeted bispecific antibody zanidatamab will be the first of its class to hit the market for biliary tract cancer. The drug, to be marketed as Ziihera, won accelerated approval in November for previously treated patients who scored high on an immunohistochemistry test measuring HER2 expression. A Phase 3 confirmatory trial is underway in the first-line setting, which executives say could support both a full approval in the second-line setting and a supplemental application for frontline patients. — Nicole DeFeudis Active ingredient: acoramidis Indication: Cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis in adults to reduce cardiovascular death and cardiovascular-related hospitalization Snapshot : Attruby, approved in November, is expected to compete with Pfizer’s Vyndaqel franchise and Alnylam’s Amvuttra, should the FDA greenlight the latter ahead of its March 23 decision date. BridgeBio and Alnylam also traded blows as the drugs moved through the clinic (Vyndaqel was approved in 2019). Notably, Attruby’s label said the drug reduces the likelihood of both hospitalization and death, putting it on the same ground as Vyndaqel. — Max Gelman Active ingredient: zenocutuzumab-zbco Indication: Pancreatic adenocarcinoma or NSCLC that harbors NRG1 gene fusions and are advanced unresectable or metastatic Snapshot : The new cancer drug marked the first commercial product for Merus. But it will be Partner Therapeutics that actually commercializes the product in the US, after paying for rights in 2024. — Max Bayer Active ingredient: cosibelimab-ipdl Indication: Adults with locally advanced or metastatic cutaneous squamous cell carcinoma who cannot receive curative surgery or radiation Snapshot : A new PD-L1 drug in 2024? Apparently so. Checkpoint Therapeutics won approval late in the year for its checkpoint inhibitor cosibelimab, which will be branded as Unloxcyt. The drug won approval for a form of advanced skin cancer, where Merck’s Keytruda and Regeneron’s Libtayo are already approved. Checkpoint was part of an earlier wave of companies that wanted to disrupt how the blockbuster class of cancer drugs are priced by selling their treatments at a discount compared to currently approved options, but those aspirations have failed to become reality. Checkpoint now says it believes its drug can be priced “at or near parity with other approved anti-PD-(L)1 therapies,” and the company is seeking a partner or business deal before launching the drug commercially. — Lei Lei Wu Active ingredient: crinecerfont Indication: To be used together with glucocorticoids to control androgen levels in adults and pediatric patients 4 years and older with classic congenital adrenal hyperplasia Snapshot : Neurocrine’s second approved product marked the first new drug for congenital adrenal hyperplasia in 70 years. It also brings some of the work of the company’s late founder, Wylie Vale, to fruition: Vale, who died in 2012, identified the compound that this drug works to block. Analysts at William Blair believe the drug could reach nearly $1.5 billion in peak sales by 2030. — Max Bayer Active ingredient: ensartinib Indication: Adult patients with anaplastic lymphoma kinase-positive locally advanced or metastatic non-small cell lung cancer who have not previously received an ALK inhibitor Snapshot : Xcovery won its first-ever FDA approval for a first-line treatment of adults with anaplastic lymphoma kinase (ALK)-positive locally advanced or metastatic non-small cell lung cancer. The approval was based on a trial of 290 patients who had not previously received an ALK-targeted therapy, and FDA said Ensacove demonstrated a statistically significant progression-free survival improvement compared to Pfizer’s Xalkori. Xcovery is also testing Ensacove in other cancers. — Zachary Brennan Active ingredient: remestemcel-L-rknd Indication: Steroid-refractory acute graft-versus-host disease in pediatric patients 2 months and older Snapshot : Ryoncil took a winding path to approval, including two rejections and concerns from regulators about the design of its single-arm trial. An approval came more than four years after regulators accepted Mesoblast’s first application. But CEO Silviu Itescu believes it was worth the wait. Ryoncil is now the first mesenchymal stromal cell therapy to be approved by the FDA. It will be available for pediatric patients 2 months and older with acute graft-versus-host disease, a complication of allogeneic transplant in which donor cells attack the recipient. — Nicole DeFeudis Active ingredient: olezarsen Indication: As an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome Snapshot : Ionis’ antisense oligonucleotide olezarsen is the first approved treatment for familial chylomicronemia syndrome, a rare metabolic disorder that prevents the body from breaking down fats called triglycerides. The approval marks an important transition for Ionis, as Tryngolza will be the company’s first independent launch following years of partnerships. While FCS affects few people in the US, CEO Brett Monia hopes that a potential label expansion to a broader condition called severe hypertriglyceridemia could make Tryngolza a blockbuster. — Nicole DeFeudis Active ingredient: concizumab-mtci Indication: For routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A or B with inhibitors Snapshot : The anti-tissue factor pathway inhibitor Alhemo finally came good for Novo Nordisk at the tail end of 2024 following its rejection by the FDA in May 2023. It is the first daily subcutaneous treatment for hemophilia A or B patients with inhibitors — antibodies that neutralize clotting factors — to be delivered via a pen injector. It will compete with Pfizer’s similar weekly shot Hympavzi, but could reach more patients since Hympavzi is only approved for hemophilia A and B patients without inhibitors. — Elizabeth Cairns Active ingredient: vanzacaftor, tezacaftor, and deutivacaftor Indication: Cystic fibrosis in patients aged 6 years and older who have at least one F508del mutation or another responsive mutation in the CFTR gene Snapshot : Alyftrek is Vertex’s “vanza triple” combination treatment for cystic fibrosis, a modulator of the CFTR gene. It’s the fifth such therapy Vertex has pushed across the finish line, but it comes with a boxed warning for potential drug-induced liver injury and failure. The treatment succeeded in its Phase 3 trials earlier this year, and Vertex used a priority review voucher to reduce the FDA’s review time to six months. — Max Gelman

▎药明康德内容团队编辑 2024年即将过去。在这一年里，全球生物医药产业见证了大量创新涌现，也为全球病患带来了全新的治疗机遇。辞旧迎新之际，让我们用A到Z这26个字母，和各位读者朋友们一同回顾今年广受关注的一些内容，以此总结2024年的全球生物医药产业。 Artificial Intelligence  2024 SUMMARY  今年10月，诺贝尔基金会宣布将今年的诺贝尔化学奖颁发给了三位研究领域与人工智能相关的科学家。其中，被誉为“AlphaFold之父”的DeepMind公司Demis Hassabis博士和John Jumper博士因为蛋白质结构预测获奖，而华盛顿大学的David Baker教授因计算蛋白设计获此殊荣。他们开发的AI解决方案成功解决了50年来蛋白质结构预测领域的重大挑战，加速了生物医药领域的科学发现。 人工智能在药物发现和开发领域的进展可谓日新月异。今年，Isomorphic Labs与DeepMind共同推出新一代AI生物分子结构模型AlphaFold 3。它能够针对多种蛋白质、DNA、RNA和小分子配体之间的相互作用进行复合物结构预测。这一技术能增强针对难以靶向靶点的药物理性设计，有望为未来药物发现带来崭新的一页。 Bi-Specific Antibodies  2024 SUMMARY  双特异性抗体在血液癌症领域已经得到广泛应用，多款T细胞接合器已经获得FDA的批准。而在治疗实体瘤方面，靶向两种不同信号通路的双特异性抗体也表现出卓越的疗效。今年9月，Summit Therapeutics在2024年世界肺癌大会（WCLC 2024）公布的3期临床试验数据显示，潜在“first-in-class”双特异性抗体ivonescimab与获批重磅免疫检查点抑制剂相比，作为一线疗法，显著降低了PD-L1阳性晚期非小细胞肺癌（NSCLC）患者疾病进展或死亡的风险。行业媒体Fierce Pharma发表的文章表示，这一结果具有改变NSCLC一线标准治疗的潜力。目前，同时靶向PD-1/PD-L1信号通路和VEGF信号通路的双特异性抗体已经成为多家大型药企布局的重要治疗模式之一。 CAR-T  2024 SUMMARY  今年的多项研究结果显示了CAR-T细胞疗法在治疗自身免疫性疾病方面的潜力。今年2月，《新英格兰医学杂志》发表了一项具里程碑意义的研究成果。首批接受CAR-T疗法治疗的15位严重自身免疫性疾病患者，在经过中位15个月的随访后，均显示出持续的病情缓解或症状大幅改善，并已停止所有免疫抑制与抗炎药物的使用。 多家开发CAR-T细胞疗法的公司也在今年宣布CAR-T疗法治疗自身免疫性疾病的研发项目，治疗包括红斑狼疮、重症肌无力、多发性硬化等多种疾病类型。 DMD  2024 SUMMARY  杜氏肌营养不良（DMD）患者在今年迎来两款让广泛患者受益的创新疗法。今年3月，美国FDA批准口服药物Duvyzat（givinostat）上市，用于治疗六岁及以上DMD患者。这是首个获批可用于治疗携带任何DMD基因变异患者的非类固醇疗法。此外，Sarepta Therapeutics与罗氏联合开发的基因疗法Elevidys在6月获得FDA的完全批准，治疗可行走的4岁以上DMD患者。 Epigenetic Editing  2024 SUMMARY  表观遗传编辑系统能够在不改变DNA序列的情况下，利用细胞的天然调节系统精准、持久地控制多个基因的表达水平。今年年初，基因编辑先驱张锋博士联合创建了致力于开发表观遗传编辑系统的Moonwalk Biosciences公司，并完成5700万美元的种子轮和A轮融资。今年在《自然》杂志上发表的论文和Chroma Medicine公司发布的临床前研究均显示表观遗传编辑能够通过一次治疗，长期降低PCSK9基因的表达，显示出这一技术的持久性。 FDA New Approvals  2024 SUMMARY  ▲今年FDA药物评价和研究中心批准的新药中，小分子药物占比约60%，稳居第一（药明康德内容团队制图，数据截至2024年12月20日） 截至12月20日，美国FDA旗下药物评价和研究中心（CDER）已经批准了50款新药。CDER批准新药中小分子药物占比约60%，显示出其旺盛的生命力。近5年来，多肽和核酸类药物在获批新药中的占比平均约为10%，显示这一治疗模式也成为新药的重要来源。 GLP-1  2024 SUMMARY  GLP-1药物不但在治疗2型糖尿病和肥胖症方面显示出卓越的效果，而且在临床试验中展示了治疗其它疾病的广泛潜力。12月20日，礼来（Eli Lilly and Company）公司的Zepbound获得美国FDA的批准，成为首个获批用于治疗中重度阻塞性睡眠呼吸暂停（OSA）肥胖患者的处方药。GLP-1药物还在治疗代谢功能障碍相关脂肪性肝炎（MASH），延缓心衰恶化，治疗化脓性汗腺炎等疾病的临床试验中展现了卓越的效果。 HIV Prevention  2024 SUMMARY  日前，《科学》杂志公布了2024年度的科学突破。每半年注射一次的长效HIV疗法lenacapavir当选年度突破。今年7月发布于《新英格兰医学杂志》的临床试验结果显示，lenacapavir在女性中作为暴露前预防（PrEP）疗法，显示出100%的有效性。 相关阅读：有效性近95%！多项长效HIV疗法开发中，艾滋病患者有望迎来功能性治愈的未来 Innovation  2024 SUMMARY  截至12月20日，今年CDER批准的创新疗法中，约62%源于生物技术公司，与近5年来最高点持平。这也与产业近年来的分析一致，表明生物技术公司已成为重要创新来源。近年来，更多中小型生物技术公司有意愿推动创新疗法开发直至获批上市，而赋能平台公司的助力，也让这一策略的可行性大幅度提高。 JAK Inhibitors  2024 SUMMARY  今年7月，美国FDA批准Sun Pharmaceutical开发的口服JAK1/2选择性抑制剂Leqselvi（deuruxolitinib）用于治疗成人重度斑秃。在临床试验的第8周，Leqselvi相比安慰剂便显著提高了患者实现SALT评分≤20（头皮毛发覆盖率≥80%）的比例，展现出明确的疗效。 Kisunla  2024 SUMMARY  今年7月，美国FDA批准礼来的抗β淀粉样蛋白（Aβ）抗体Kisunla（donanemab）上市，用于治疗早期症状的阿尔茨海默病（AD）成年人。根据新闻稿，每月一次的Kisunla是首个在清除淀粉样斑块后可停止治疗的疗法，预计能降低治疗成本并减少输液次数。 Lifileucel  2024 SUMMARY  美国FDA在2月加速批准Iovance Biotherapeutics的肿瘤浸润淋巴细胞（TIL）疗法Amtagvi（lifileucel），用于治疗晚期黑色素瘤。根据新闻稿，Amtagvi是首款获批的TIL疗法，也是首款获批治疗实体瘤的T细胞疗法，是细胞疗法的又一里程碑。 MASH  2024 SUMMARY  Madrigal Pharmaceuticals公司所开发的Rezdiffra（resmetirom）在3月获FDA批准，用于联合饮食控制和运动，治疗患有中重度肝纤维化（F2至F3期）的代谢功能障碍相关脂肪性肝炎成人患者。根据新闻稿，这是获FDA批准的首款MASH疗法，标志着该领域的一项重要里程碑。 Niemann-Pick Disease Type C  2024 SUMMARY  C型尼曼匹克病（NPC）患者在今年一周内迎来首款和第二款FDA批准的疗法。9月20日， Zevra Therapeutics公司开发的“first-in-class”细胞热休克反应共诱导剂Miplyffa（arimoclomol）获得FDA批准，与酶抑制剂miglustat联用，治疗成人和2岁及以上儿童的NPC相关神经系统症状。不到一周，美国FDA批准由IntraBio公司开发的Aqneursa（levacetylleucine）上市，为NPC患者带来更多治疗选择。 Ohtuvayre  2024 SUMMARY  Verona Pharma在6月宣布其“first-in-class”磷酸二酯酶3/4（PDE3/4）抑制剂Ohtuvayre（ensifentrine）获FDA批准，用于成人慢性阻塞性肺病（COPD）维持治疗。新闻稿指出，这是一款兼具支气管扩张与非类固醇抗炎作用的首个吸入式COPD疗法，也是20多年来首个具有新机制的COPD吸入式维持治疗药物。 Protein Degraders  2024 SUMMARY  靶向蛋白降解已经成为新药开发的重要热门领域之一。今年多项科学研究为靶向蛋白降解提供更多工具和技术平台，进一步扩展可降解蛋白的范围。例如，刘如谦教授团队今年3月在《科学》杂志发表研究，介绍了一种通过特定分子胶调控的蛋白降解系统，该系统理论上可以在生物体内有选择性地调控任意内源性蛋白的表达。 诺奖得主David Baker和Carolyn Bertozzi教授合作在《自然》杂志上描述了一种全新的蛋白降解药物类型。这种名为内吞触发结合蛋白（EndoTag）的蛋白通过与细胞膜表面的受体相结合，促进内吞作用的发生，从而介导蛋白降解。 Quantum Computing  2024 SUMMARY  今年12月，谷歌量子人工智能研究团队在《自然》发表了一项里程碑式的突破，其量子芯片Willow展现了创新的纠错能力，为扩展量子芯片的性能，完成大规模容错量子计算提供了突破口。在这项研究中，该芯片可以在五分钟内完成当今最快的超级计算机需要花费1025年才能完成的计算课题。虽然量子计算技术仍然处于早期阶段，但是科学家们已经开始展望将其用于药物设计和开发方面的前景。 Radioligand Therapy  2024 SUMMARY  放射性配体疗法（RLT）通过将放射性同位素与靶向癌细胞的配体连接在一起，将放射性同位素靶向递送到癌症细胞附近，在杀死癌细胞的同时，最小化对周围健康组织的损伤。这一领域近年来受到广泛关注，不但多家新锐公司成立，大药企也通过收购和合作在这一领域纷纷布局。比如诺华公司在今年斥资超过17亿美元收购了Mariana Oncology。阿斯利康（AstraZeneca）则收购了Fusion Pharmaceuticals。百时美施贵宝（Bristol Myers Squibb）在今年2月完成对RayzeBio的收购。 相关阅读：近百亿美元加码布局！这一抗癌模式为何得到诸多大药企青睐 Schizophrenia  2024 SUMMARY  百时美施贵宝的Cobenfy（xanomeline和trospium chloride，即KarXT）口服胶囊于9月获FDA批准，用于治疗成人精神分裂症患者。FDA新闻稿指出，这是首个靶向胆碱能受体的抗精神病药物，突破了长期以来多巴胺受体为主的治疗模式，标志着数十年来精神分裂症治疗领域的新突破。 TCR-T  2024 SUMMARY  Adaptimune Therapeutics的工程化T细胞疗法Tecelra（afami-cel）在8月获FDA批准，用以治疗既往接受过化疗的某些HLA类型的晚期MAGE-A4+滑膜肉瘤成人患者。根据新闻稿，Tecelra为首款用于治疗实体瘤的工程化T细胞疗法，也是十多年来治疗滑膜肉瘤的首个有效疗法。 Undruggable  2024 SUMMARY  随着蛋白降解技术，共价药物，别构药物开发领域的进展，“不可成药”靶点已经不再遥不可及。根据药明康德内容团队统计，大型药企的研发管线中至少有39款临床期在研疗法针对难以成药靶点或采用创新靶向技术。此外，基于生成式人工智能技术平台进行新药发现和开发的新锐公司不断涌现，这些公司的平台可以根据靶点的详细特征，从头设计与它们相结合的小分子或蛋白，为攻克难于成药靶点提供了新策略。例如，Nabla Bio今年完成2600万美元A轮融资，并已经与阿斯利康、百时美施贵宝和武田（Takeda）等大型医药公司达成战略合作，预付款、里程碑付款总计可达5.5亿美元。 相关阅读：AI蛋白设计领域，这7家公司值得关注！ Venture Capital  2024 SUMMARY  虽然今年的投融资市场整体尚未完全复苏，但是在早期投融资领域，多家知名风投对新锐公司进行了大额投资。根据药明康德内容团队的统计，在生物医药领域，至少9家新锐公司完成超过2亿美元的A轮或B轮融资。这些获得大额融资的新锐公司中，多家公司专注于免疫和炎症疾病领域的新药开发，例如Mirador Therapeutics，Candid Therapeutics等。致力于开发肥胖和代谢类疾病疗法的公司也获得风投公司的青睐，例如Metsera今年完成2.9亿美元的A轮融资和2.15亿美元的B轮融资，而Kailera Therapeutics公司今年完成4亿美元的A轮融资。 WVE-006  2024 SUMMARY  今年10月，Wave Life Sciences宣布，在研RNA编辑候选疗法WVE-006在临床试验中成功编辑α-1抗胰蛋白酶缺乏症（AATD）患者的基因，完成临床机制验证。WVE-006是首个进入临床开发的RNA编辑疗法，而这一试验结果是RNA编辑疗法在人体当中完成首次机制验证，为开发RNA编辑这一创新治疗模式迈出了重要一步。 Xenotransplantation  2024 SUMMARY  利用基因工程改造的猪器官替代人体器官进行异种器官移植在今年取得多项突破。今年3月，麻省总医院（MGH）和eGenesis公司共同宣布，MGH的医生团队成功将eGenesis公司通过CRISPR基因编辑技术改造的猪肾脏移植到一位62岁的终末期肾病患者体内。这是世界上首次成功将基因编辑的猪肾脏移植到活患者体内的案例。该患者恢复良好并成功出院，虽然在两个月后因为意外的心脏事件而去世，但是研究人员的调查显示，他去世时并没有出现对猪肾脏的免疫排斥反应。 今年12月，纽约大学Langone健康中心宣布，一名女性患者在接受United Therapeutics旗下的Revivicor公司基因编辑改造的猪肾移植手术后，停止接受透析，并且健康状况显著改善，已经获准出院。这些进展有望促进猪肾脏人体移植临床试验的开展，为变革器官移植领域带来希望。 Yorvipath  2024 SUMMARY  FDA在8月批准Ascendis Pharma的Yorvipath（palopegteriparatide），用于治疗成人甲状旁腺功能减退症。根据新闻稿，Yorvipath是首款获FDA批准用于治疗成人甲状旁腺功能减退患者的药物。 Zevtera  2024 SUMMARY  今年4月，Basilea Pharmaceutica开发的Zevtera（ceftobiprole medocaril sodium）获FDA批准用于治疗三种适应症，包括金黄色葡萄球菌菌血症（SAB）、急性细菌性皮肤及结构感染（ABSSSI）和社区获得性细菌性肺炎（CABP），为应对抗微生物耐药性（AMR）提供了新的工具。药明康德内容团队也在今年“世界提高微生物药物耐药性认识周”期间，发表了针对AMR的系列文章，呼吁全球协作，共同抗击这一日益严重的健康威胁。 相关阅读：死亡人数即将反超癌症，药物研发却严重滞后，如何应对这一全球健康威胁？ 一切过往，皆为序章。站在2024年的尾声，让我们共同期待明年有更多创新涌现，将更多好药新药更快带给全球病患，满足未竟的医疗需求！  喜欢我们文章的朋友点个“在看”和“赞”吧，不然微信推送规则改变，有可能每天都会错过我们哦~ 免责声明 “汇聚南药”公众号所转载文章来源于其他公众号平台，主要目的在于分享行业相关知识，传递当前最新资讯。图片、文章版权均属于原作者所有，如有侵权，请在留言栏及时告知，我们会在24小时内删除相关信息。 信息来源：原创 药明康德 往期推荐 本平台不对转载文章的观点负责，文章所包含内容的准确性、可靠性或完整性提供任何明示暗示的保证。