Regeneron Pharmaceuticals, Inc.

EMPAVELI's unique mechanism of targeting C3 broadens its applicability beyond PNH, including other complement-driven diseases like geographic atrophy and immune complex-mediated diseases. With a high unmet need in these areas and limited competition, EMPAVELI's market is poised for steady growth. LAS VEGAS, March 18, 2025 /PRNewswire/ -- DelveInsight's " EMPAVELI Market Size, Forecast, and Market Insight Report" highlights the details around EMPAVELI, a complement inhibitor indicated to treat adult patients with PNH. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of EMPAVELI. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Apellis Pharmaceutical's EMPAVELI (pegcetacoplan) Overview EMPAVELI (pegcetacoplan) is a targeted inhibitor of C3 designed to control excessive complement activation—a key immune process that can contribute to the development and progression of serious diseases. Pegcetacoplan binds to complement protein C3 and its activation fragment C3b, helping to regulate the cleavage of C3 and the production of downstream effectors involved in complement activation. In PNH, extravascular hemolysis (EVH) is driven by C3b opsonization, while intravascular hemolysis (IVH) results from the formation of the membrane attack complex (MAC). By acting early in the complement cascade, pegcetacoplan controls both C3b-driven EVH and terminal complement-mediated IVH. EMPAVELI is approved for treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH). The US FDA has granted pegcetacoplan Priority Review and Fast Track Designation (FTD) for the treatment of PNH. EMPAVELI reported US net product revenue of $23.4 million in the fourth quarter and $98.1 million for the full year of 2024. Currently, EMPAVELI is being evaluated in different phases of development for C3G & IC-MPGN, HSCT-TMA, FSGS, and DGF. Learn more about EMPAVELI projected market size for PNH @ EMPAVELI Market Potential Paroxysmal nocturnal hemoglobinuria (PNH) is a rare condition characterized by a triad of symptoms: intravascular hemolysis, thromboembolic events, and cytopenia. The disease presents differently among patients, making it difficult to classify based on typical clinical patterns due to its unpredictable nature. In 2023, there were approximately 12,000 diagnosed cases of PNH in the 7MM, with projections suggesting this number could rise to around 13,000 by 2034. Until 2007, PNH was a life-threatening disease with no effective treatment for hemolysis and thrombosis, which were the primary causes of death. However, the introduction of the anti-C5 agent eculizumab over the past decade marked a major breakthrough, significantly reducing hemolysis, decreasing the need for transfusions, and lowering the risk of thrombosis. The current standard of care for PNH involves complement inhibition therapy, with FDA-approved drugs like SOLIRIS, ULTOMIRIS, EMPAVELI, FABHALTA, VOYDEYA, and PIASKY being considered the gold standard treatments. According to DelveInsight, the total PNH market size in the 7MM was valued at USD 1.4 billion in 2023 and is expected to grow to about USD 2.5 billion by 2034. This growth is driven by advances in understanding disease mechanisms, leading to improved diagnostics and therapeutics, along with an increasing number of cases and the introduction of new treatments during the forecast period. Discover more about the paroxysmal nocturnal hemoglobinuria market in detail @ Paroxysmal Nocturnal Hemoglobinuria Market Report Emerging Competitors of EMPAVELI Only a few companies are currently working in the paroxysmal nocturnal hemoglobinuria market. Emerging PNH therapies include Pozelimab (REGN3918) + Cemdisiran (Regeneron Pharmaceuticals), OMS906 (Omeros), NM8074 (ruxoprubart) (NovelMed), and others, reflecting a dynamic evolution in treatment strategies. In December 2024, Omeros Corporation announced that two posters on zaltenibart (OMS906), its investigational MASP-3 inhibitor and a key activator of the alternative complement pathway, were presented yesterday at the 66th Annual Meeting of the American Society of Hematology (ASH) in San Diego. The posters focused on zaltenibart's use in treating paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder, and highlighted positive Phase II clinical data and clinical pharmacology analyses that support dose selection for the upcoming Phase III trials in PNH. Enrollment for the Phase III trials is expected to begin in early 2025. To know more about the number of competing drugs in development, visit @ EMPAVELI Market Positioning Compared to Other Drugs Key Milestones of EMPAVELI In October 2023, Apellis Pharmaceuticals, Inc. announced that the FDA had approved the EMPAVELI Injector. In May 2021, Apellis Pharmaceuticals, Inc. announced that the FDA has approved EMPAVELI (pegcetacoplan), the first and only therapy targeting C3 for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). In October 2020, Apellis Pharmaceuticals and Swedish Orphan Biovitrum AB announced a strategic collaboration to accelerate the advancement of systemic pegcetacoplan. In February 2019, Apellis Pharmaceuticals announced that the FDA has awarded Fast Track designation to APL-2, the company's innovative complement factor C3 inhibitor, for treating patients with paroxysmal nocturnal hemoglobinuria (PNH). Discover how EMPAVELI is shaping the PNH treatment landscape @ EMPAVELI Paroxysmal Nocturnal Hemoglobinuria EMPAVELI Market Dynamics EMPAVELI (pegcetacoplan) is a targeted complement inhibitor developed by Apellis Pharmaceuticals for the treatment of PNH. Approved by the FDA in May 2021, EMPAVELI became the first and only C3 inhibitor available for PNH, providing a new mechanism of action compared to existing therapies like C5 inhibitors (eculizumab and ravulizumab). By targeting C3 rather than C5, EMPAVELI addresses both intravascular and extravascular hemolysis, offering broader control over the disease and reducing the need for red blood cell transfusions in patients who remain anemic despite C5 inhibition. This differentiation has positioned EMPAVELI as a compelling alternative for patients with suboptimal responses to C5 inhibitors. The market for EMPAVELI is driven by the growing prevalence of PNH, increased awareness of complement pathway disorders, and improved diagnostic capabilities. The PNH market was historically dominated by C5 inhibitors such as Alexion's SOLIRIS (eculizumab) and ULTOMIRIS (ravulizumab), which set a high pricing benchmark and established market acceptance for premium-priced orphan drugs. EMPAVELI's entry has introduced competition, particularly for patients who experience breakthrough hemolysis or insufficient control on C5 therapy. Its ability to offer a more comprehensive mechanism of action and potential for improved patient outcomes has driven adoption, although switching patients from established therapies presents a challenge due to physician familiarity and the entrenched market position of C5 inhibitors. Pricing and reimbursement dynamics also play a significant role in EMPAVELI's market performance. As a high-cost orphan drug, EMPAVELI's adoption is influenced by payer coverage and reimbursement policies, especially in markets with strict cost-control measures. Apellis has strategically positioned EMPAVELI through patient assistance programs and value-based agreements to facilitate market access. Additionally, the subcutaneous administration of EMPAVELI (versus intravenous administration for C5 inhibitors) enhances patient convenience, supporting patient preference and potentially improving adherence. Competitive pressures are also evolving as other complement inhibitors targeting C3, C5, and alternative pathways are under development. For example, Novartis and AstraZeneca are advancing next-generation C5 inhibitors with extended dosing intervals, and other biotech firms are exploring factor D and MASP-2 inhibitors, which could further fragment the complement inhibitor landscape. However, EMPAVELI's first-mover advantage in the C3 inhibition space and its differentiated clinical profile provide a strong foothold in the market. Expansion into additional indications beyond PNH, such as cold agglutinin disease (CAD) and geographic atrophy (GA), could further strengthen EMPAVELI's market position and drive future growth. Dive deeper to get more insight into EMPAVELI's strengths & weaknesses relative to competitors @ EMPAVELI Market Drug Report Table of Contents Related Reports Paroxysmal Nocturnal Hemoglobinuria Market Paroxysmal Nocturnal Hemoglobinuria Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key PNH companies including Hoffmann-La Roche, Alexion Pharmaceuticals, Novartis, Regeneron Pharmaceuticals, BioCryst Pharmaceuticals, among others. Paroxysmal Nocturnal Hemoglobinuria Pipeline Paroxysmal Nocturnal Hemoglobinuria Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key paroxysmal nocturnal hemoglobinuria companies, including Biocad, AKARI Therapeutics, Amgen, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, among others. Aplastic Anemia Market Aplastic Anemia Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key aplastic anemia companies, including Pfizer, BioLineRx Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals, among others. Aplastic Anemia Pipeline Aplastic Anemia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key aplastic anemia companies, including Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

The end of the U.S. patent runway is nearing for 10 industry blockbusters in 2025. While each year features high-profile losses of exclusivity in the pharma industry, this year's list is something of a doozy.For one, Johnson & Johnson's Stelara is already facing off against several biosimilars, and several more are yet to come as the year plays out. With $6.72 billion in U.S. sales in 2024, the drug is the largest U.S. loss of exclusivity since AbbVie's megablockbuster Humira tumbled over the patent cliff in early 2023. Johnson & Johnson had previously warned of a "late 2023" patent cliff for Stelara, but the company was able to squeeze more life out of its key immunology moneymaker through a series of legal settlements.Johnson & Johnson's not alone in losing U.S. market exclusivity on a key sales driver this year. Also facing ongoing or future declines for their top revenue generators are Regeneron with eye drug Eylea, Amgen with bone medicines Prolia/Xgeva and Novartis with heart failure therapy Entresto. Eylea already faces its first U.S. biosimilar after Amgen launched Pavblu in November. Fierce Pharma chose to include Eylea in this report—despite the biosimilar launch happening last year—because the commercial situation is still playing out for Regeneron and to not exclude a key development in an important market.Amgen, for its part, collected $4.39 billion in revenue from its key bone medicines Prolia and Xgeva last year. While the company has built its own impressive biosimilar business over the years, it'll have to play an unfamiliar role and watch copycat drugs eat away at the market for its originators starting in late May and early June.Novartis, meanwhile, has warned of a "mid-2025" loss of U.S. exclusivity for Entresto as a key combination patent nears its expiration this summer. Besides Entresto, Novartis has two other big-selling products on this list in Promacta and Tasigna.Moving down the list, readers will likely notice two high-profile drugs from AstraZeneca. Rare disease medicine Soliris, picked up in AZ's buyout of Alexion, is set to face its first biosimilar from Amgen sometime in the second quarter. And Brilinta, a cardiometabolic drug once pegged to be a key blockbuster but that never quite met expectations, is also running out of its U.S. patent protections in the near future.For one measure of the high-profile nature of the drugs on this list, look no further than last year's version of the report. With $1.45 billion in prior-year U.S. sales, Bristol Myers Squibb's Sprycel led the 2024 group. But, if compared against the 2025 class of patent expirations, Sprycel would rank No. 6. There are always uncertainties in compiling this report due to the unpredictable nature of patent litigation and regulatory interactions. This year, the drug with the most apparent uncertainty is J&J's Simponi and Simponi ARIA. That medicine's inclusion hinges on a potential fourth-quarter approval—and launch—of Teva and Alvotech's AVT05. We opted to include the medicine because a key patent has already expired, and a reputable biosimilar team is marching through the regulatory process.To compile this report, Fierce Pharma parsed company filings, presentations and conference call transcripts. We also used published research from pharmacy benefit manager OptumRx and healthcare services firm Cardinal Health. The report ranks the top U.S. losses of exclusivity based on U.S. sales from the prior year.

Welcome back to Endpoints Weekly. Our reporters tracked a flurry of deals and data readouts this week, plus updates on the Trump administration’s health agency picks. Let’s dive in. The Trump administration pulled its nomination for CDC candidate Dave Weldon hours before he was set to appear before the Senate HELP committee on Thursday. The move came as a surprise following what’s been a relatively easy path for other healthcare appointments. Endpoints’ Max Bayer has the details below. Keep reading for a summary of this week’s deals, including Roche and Zealand Pharma’s massive obesity partnership and Bristol Myers Squibb’s acquisition of 2seventy bio. And Elizabeth Cairns has the latest on Pfizer and Arvinas’ breast cancer candidate that extended progression-free survival in certain patients in a Phase 3 trial, but not in the overall population. The companies still plan to file for approval, she reported. Thanks for reading, and have a great weekend! — Nicole DeFeudis 🏛️The Trump administration pulled its nomination for Dave Weldon to lead the CDC on Thursday, hours before his Senate confirmation hearing was scheduled to begin. Weldon, a former Florida congressman, has been sympathetic to concerns about vaccines in the past and was seen as an ally of HHS Secretary Robert F. Kennedy, Jr.’s push to reopen long-settled questions about vaccine safety. The debunked link between vaccines and autism came up several times during the confirmation hearings for Kennedy, as well as for Trump’s NIH and FDA nominees. Two other healthcare nominees are advancing to the full Senate: FDA nominee Marty Makary and NIH nominee Jay Bhattacharya. The Senate HELP committee voted 14-9 in favor of Makary, and 12-11 for Bhattacharya. Mehmet Oz, Trump’s pick to lead CMS, sailed through a Senate confirmation hearing on Friday. The former surgeon and TV personality was questioned about the high cost of healthcare, potential cuts to Medicaid and more. Most of his responses seemed to satisfy legislators. When asked whether he would continue to defend Medicare drug price negotiations under the IRA, Oz responded: “It’s the law. I’m going to defend it and use it.” Meanwhile, the Trump administration must reinstate probationary government employees that it fired without proper notice, according to a court order. A Maryland federal judge granted a temporary restraining order against the government in a legal challenge brought by 20 states over mass layoffs. The order applies to over a dozen federal agencies, including HHS, that were named defendants in the suit. It restores the status quo for 14 days while the court considers a longer injunction. 📉 Pfizer and Arvinas are partnered on protein degrader technology that is aiming to change the treatment paradigm of certain cancers. But the pair ran into a stumbling block this week when they reported that their vepdegestrant program did not extend progression-free survival rates in the overall population of a Phase 3 breast cancer study. Arvinas’ stock price fell more than 50% on the news. The trial was considered the first big test of a class of drugs called proteolysis-targeting chimeras, or PROTACs, and Pfizer had high hopes for the program after paying $1 billion upfront to partner with Arvinas in 2021. Researchers were testing vepdegestrant as a monotherapy in estrogen receptor-positive, HER2-negative metastatic breast cancer. Patients were in the second-line setting, having previously received CDK4/6 inhibitors and hormone therapy. Vepdegestrant isn’t completely dead, as Pfizer and Arvinas will still seek approval. Despite its apparent lack of effect in the overall trial population, the drug cut the risk of progression or death by at least 40% (the companies did not provide the exact figure) compared to the hormone therapy Faslodex in patients with mutated estrogen receptor genes. But it may not get as broad a label as investors and executives may have wanted. 🧬 Beam Therapeutics, meanwhile, saw some good news when it reported this week that an experimental base editing treatment helped lower levels of mutant proteins — and increase levels of corrected proteins — in nine people with a rare lung disease. The one-time therapy reduced levels of mutant proteins by 11% and 38% in the first two dose levels. And, in one patient at the highest dose, the treatment reduced levels of mutant protein by 78%. Beam is still deliberating whether it will test higher doses. The company claims it’s the first clinical results using base editing to directly correct a disease-causing mutation. It’s also Beam’s first trial results using in vivo base editing, which is considered to be a sort of CRISPR 2.0 with its ability to insert single-letter changes to the DNA. There were no side effects, and the protein was detected within three days of treatment. 🤝Roche and Zealand Pharma will collaborate on the biggest obesity partnership to date, with Roche ponying up $1.65 billion upfront for an amylin analog called petrelintide. Including milestone payments, the deal could be worth up to $5.3 billion. They plan to test petrelintide in combination with some of Roche’s other pipeline programs, including the GLP-1/GIP agonist CT-868, and a pill called CT-996. Zealand CEO Adam Steensberg told Endpoints News that obesity will be the first indication, “with an ambition of rapidly expanding.” Bristol Myers also got in on the action, acquiring its troubled cell therapy partner 2seventy bio, which spun out from bluebird bio, for $286 million in cash. The companies are partnered on the CAR-T therapy Abecma. 2seventy has fallen on hard times of late, having sold off most of its R&D work to Regeneron and laying off staff. BMS is buying 2seventy for $5 per share after the company peaked at $38 — but 2seventy fared better than bluebird, after SEC documents revealed its buyout offer shrunk from $100 million to $29 million. And in a smaller deal this week, Daiichi Sankyo and Johnson & Johnson both teamed up with Nosis Bio, a startup developing new ways to deliver RNA therapies to specific cells. Nosis was founded in 2021 and has only raised $13 million so far, but the two pharma deals could bring in more than $1.5 billion if all the milestones are met. Much remains unknown: Nosis did not disclose how much money it received upfront from Daiichi and J&J, nor did it say what kinds of cells or diseases the partnerships entail. A biotech advancing a once-monthly liver disease drug is exploring both an IPO and an acquisition. Boston Pharma is getting ready to launch a Phase 3 study in patients with metabolic dysfunction-associated steatohepatitis, a form of advanced fatty liver disease also known as NASH. For years, the condition was mainly treated with diet and exercise, until Madrigal Pharmaceuticals won the first approval last year. A handful of competitors are in late-stage trials, but Boston Pharma’s CEO Sophie Kornowski isn’t daunted. “I’m extremely motivated to do it well, and to do it fast,” she said. The agency said this week that it won’t allow employees who review new drug or medical device applications to take a $25,000 buyout offered to all HHS staffers. These include individuals in the CDER, CBER, CDRH, CVM, CTP and OC offices. The carveouts for staff funded by industry user fee dollars show the extent to which the FDA and HHS are not targeting drug and device reviewers in the Trump administration’s wider government staffing cuts.