factor IX

Pfizer’s Hympavzi (marstacimab) has been approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA) to treat haemophilia A or B in adult and paediatric patients. The drug has been authorised to prevent or reduce bleeding episodes in patients ages 12 years and older who weigh at least 35kg and have severe haemophilia A without factor VIII inhibitors or severe haemophilia B without factor IX inhibitors. More than 11,800 people were living with haemophilia A or B in the UK in 2023/2024, including 2,700 with severe disease. The genetic blood disorders primarily affect males and are caused by a clotting factor deficiency. Alongside episodes of excessive and prolonged bleeding, haemophilia patients can experience spontaneous bleeding in their joints and muscles without having had an injury, potentially leading to joint damage. Pfizer’s Hympavzi, given as a once-weekly subcutaneous injection using a pre-filled pen, works by reducing the amount of TFPI, a naturally occurring protein that prevents blood from clotting too much. This promotes the formation of thrombin, an enzyme critical in blood clotting. The MHRA’s decision on the drug was supported by results from the late-stage BASIS trial, in which the annualised bleeding rate for treated bleeds was 7.85 for routine factor-based prophylaxis during a six‑month observation period and 5.08 for Hympavzi prophylaxis during a 12‑month active treatment period. Seema Patel, medical director specialty care, Pfizer UK, described the authorisation as “an important step towards providing eligible patients with a new treatment option”. Hympavzi is now being evaluated by the National Institute for Health and Care Excellence and Scottish Medicines Consortium for use on the NHS. “We will continue working with reimbursement agencies across the UK with the aim of making [Hympavzi] available on the NHS,” Patel said. Haemophilia is a key therapeutic area for Pfizer and the company has a significant presence in the field. In July last year, its haemophilia B gene therapy was granted conditional marketing authorisation by the European Commission to treat adults with severe and moderately severe cases. In the same month, the company shared positive top-line results from a late-stage study of its investigational haemophilia A gene therapy giroctocogene fitelparvovec, which is being developed as part of a collaboration agreement between Pfizer and Sangamo Therapeutics.

HEMLIBRA has strong market potential due to its unique, subcutaneous prophylactic treatment for Hemophilia A, including patients with and without factor VIII inhibitors. With increasing global diagnosis rates and expanding access in emerging markets, HEMLIBRA is poised for continued growth. LAS VEGAS, April 21, 2025 /PRNewswire/ -- DelveInsight's " HEMLIBRA Market Size, Forecast, and Market Insight Report" highlights the details around HEMLIBRA, a bispecific factor IXa- and factor X-directed antibody indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ages newborn and older with hemophilia A (congenital factor VIII deficiency) with or without factor VIII inhibitors. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of HEMLIBRA. 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]. Chugai/Genentech/Roche's HEMLIBRA (emicizumab) Overview HEMLIBRA is a bispecific antibody that targets both factor IXa and factor X, two key proteins in the body's natural blood clotting process. Bridging these proteins it helps restore clotting function in individuals with hemophilia A. HEMLIBRA is used as a preventive treatment and is administered as a ready-to-use subcutaneous injection. It can be given once a week, every two weeks, or every four weeks—following an initial four-week period of weekly doses. Developed by Chugai Pharmaceutical Co., Ltd., HEMLIBRA is co-developed internationally by Chugai, Roche, and Genentech. In the U.S., Genentech markets the drug under the name HEMLIBRA (emicizumab-kxwh), with "kxwh" assigned per FDA guidance on naming biological products. HEMLIBRA can be prescribed in various dosage regimens. The initial loading dose is 3 mg/kg administered via subcutaneous injection once weekly for the first four weeks. This is followed by one of the following maintenance dosing options: 1.5 mg/kg once every week, 3 mg/kg once every two weeks, or 6 mg/kg once every four weeks The choice of maintenance dose should be guided by the healthcare provider's judgment, taking into account the potential for improved patient adherence with certain regimens. The prophylactic use of bypassing agents should be stopped the day before initiating HEMLIBRA prophylaxis. Factor VIII (FVIII) prophylaxis may be continued during the first week of HEMLIBRA treatment. Learn more about HEMLIBRA projected market size for hemophilia A @ HEMLIBRA Market Potential Hemophilia A is marked by bleeding episodes, though in mild and moderate cases, these initial episodes are often less intense, which can lead to delays in diagnosis as patients may not seek medical attention promptly. Moreover, early detection is frequently hindered by the lack of access to adequate diagnostic facilities. In 2023, the number of treated prevalent Hemophilia A cases across the 7MM was estimated at around 45,000, with projections indicating growth throughout the forecast period, according to DelveInsight. Among hemophilia A patients—whether or not they have inhibitors—those without inhibitors represent a larger portion of the population. The standard treatment approach focuses on replacing the missing clotting factor, FVIII, to support normal clot formation and minimize or prevent bleeding episodes. Initially, FVIII replacement was sourced from whole blood and plasma, but advances in biotechnology led to the development of recombinant FVIII (rFVIII) therapies, significantly enhancing disease management by offering more precise and efficient options. More recently, innovative therapies using novel mechanisms to prevent bleeding are expanding the treatment arsenal. Several approved therapies for hemophilia A include ALTUVIIIO (Sanofi/Sobi), ROCTAVIAN (BioMarin), OBIZUR (Takeda), ALHEMO (Novo Nordisk), HEMLIBRA (Chugai/Genentech/Roche), ESPEROCT (Novo Nordisk), JIVI (Bayer), WILATE (Octapharma), and ADYNOVATE (Takeda), among others. Notably, ROCTAVIAN, a gene therapy developed by BioMarin, represents a major advancement—it offers a one-time treatment by introducing functional FVIII genes into the patient's cells, potentially restoring normal clotting ability and reducing the need for regular injections. With a wholesale acquisition cost of USD 2.9 million, BioMarin has already facilitated reimbursement in Europe and administered treatment to a patient in Germany in Q2 2023 at a discounted price of approximately USD 900,000. Looking ahead, the hemophilia A treatment landscape is expected to evolve significantly from 2024 to 2034, fueled by the introduction of new therapies currently in development. According to DelveInsight, the hemophilia A market across the 7MM is projected to grow from USD 11.1 billion in 2023 at a robust CAGR through 2034. Nonetheless, factors like delayed diagnosis, limited awareness, and high treatment costs may pose barriers to market growth. Discover more about the hemophilia A market in detail @ Hemophilia A Market Report Emerging Competitors of HEMLIBRA Emerging therapies such as SPK-8011 (Roche [Spark Therapeutics]), fitusiran (Sanofi), marstacimab (Pfizer), Mim8 (Novo Nordisk), and others are being developed to provide safe and effective treatment options. Companies like GeneVentiv Therapeutics are also working on advancements like Adeno-associated Virus (AAV)-based universal gene therapy for hemophilia (GENV-HEM [AAV8.Fva]). Fitusiran is a subcutaneously administered small interfering RNA (siRNA) therapy under development for the prophylactic treatment of hemophilia A and B, regardless of the presence of inhibitors. Its mechanism involves reducing levels of antithrombin—a protein that inhibits clot formation—to enhance thrombin generation, restore hemostatic balance, and prevent bleeding episodes. In January 2018, Sanofi and Alnylam Pharmaceuticals restructured their RNAi therapeutics collaboration to better align and accelerate the development and commercialization of select treatments for rare genetic diseases. Through this revised agreement, Sanofi obtained global rights to develop and commercialize fitusiran. Marstacimab (PF-06741086) is a monoclonal IgG1 antibody that targets the Kunitz 2 domain of the Tissue Factor Pathway Inhibitor (TFPI). It is being developed to prevent or reduce bleeding events in patients with severe hemophilia A or B (defined as <1% activity of Factor VIII or IX), whether or not they have inhibitors. The FDA granted Fast Track Designation to marstacimab in September 2019 for its potential use in patients with inhibitors. In December 2023, Pfizer announced that the FDA had accepted its Biologics License Application (BLA) for marstacimab, supported by data from the Phase III BASIS study involving patients without FVIII or FIX inhibitors. BBM-H803 is Belief BioMed's first gene therapy candidate for hemophilia A and its second product to gain Investigational New Drug (IND) approval from China's National Medical Products Administration (NMPA). In December 2022, the U.S. FDA granted Orphan Drug Designation to BBM-H803. To know more about the number of competing drugs in development, visit @ HEMLIBRA Market Positioning Compared to Other Drugs Key Milestones of HEMLIBRA In June 2022, Chugai received approval in Japan for acquired Hemophilia A based on a domestic Phase III AGEHA study. In October 2018, HEMLIBRA was approved by the US FDA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors (FVIII). However, this therapy, which was co-developed by Genentech, Chugai, and Roche, was already FDA-approved in 2017 for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with FVIII inhibitors In April 2018, the US FDA granted breakthrough therapy designation to Genentech's HEMLIBRA for people with hemophilia A without factor VIII inhibitors. In October 2014, the US FDA granted orphan drug designation to HEMLIBRA for the treatment of Hemophilia A. Discover how HEMLIBRA is shaping the hemophilia A treatment landscape @ HEMLIBRA Cost HEMLIBRA Market Dynamics HEMLIBRA developed by Roche/Genentech, has significantly reshaped the hemophilia A treatment landscape since its approval. Designed as a bispecific monoclonal antibody, HEMLIBRA mimics the function of factor VIII by bridging activated factor IX and factor X, offering prophylactic treatment for patients with hemophilia A with or without factor VIII inhibitors. Its subcutaneous administration and less frequent dosing schedule (weekly, biweekly, or monthly) offer considerable convenience compared to traditional intravenous factor replacement therapies, making it a preferred option for both patients and caregivers. The market dynamics for HEMLIBRA have been largely influenced by its clinical efficacy, improved patient compliance, and strong safety profile. Since its launch, it has rapidly gained market share, especially among inhibitor patients where treatment options were limited and complex. In non-inhibitor patients as well, the shift away from frequent IV infusions has led to strong adoption rates, allowing HEMLIBRA to cannibalize traditional recombinant factor VIII therapies. Furthermore, its entry has pressured pricing and market share dynamics across the hemophilia therapeutic space, triggering strategic shifts by legacy players like Takeda, Sanofi, and Bayer. However, HEMLIBRA's market leadership is being challenged by the evolving competitive landscape. The emergence of gene therapies, which offer the potential for long-term or even curative outcomes, introduces a new dimension to treatment decisions. While some gene therapies are still in early stages of adoption and face challenges related to durability, safety, and patient eligibility, they represent a looming threat to the long-term dominance of HEMLIBRA, particularly in younger, eligible patient populations. Going forward, the market trajectory for HEMLIBRA will depend on several factors: expanding label indications (such as pediatric use), long-term safety and efficacy data, continued pricing negotiations with payers, and how effectively Roche can defend its position against next-generation therapies. Additionally, with increasing emphasis on patient-centric outcomes and value-based care, HEMLIBRA's ability to demonstrate long-term cost-effectiveness versus both traditional and novel competitors will be key to sustaining its market share. Dive deeper to get more insight into HEMLIBRA's strengths & weaknesses relative to competitors @ HEMLIBRA Market Drug Report Table of Contents Related Reports Hemophilia A Market Hemophilia A 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 hemophilia A companies, including BioMarin Pharmaceutical, Roche (Spark Therapeutics), ApcinteX, Novo Nordisk, Sanofi (Genzyme), Alnylam Pharmaceuticals, Pfizer, Sangamo Therapeutics, Bayer, Ultragenyx Pharmaceutical, among others. Hemophilia A Pipeline Hemophilia A Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key hemophilia B companies, including UniQure Biopharma B.V., CSL Behring, Pfizer, Spark Therapeutics, Genzyme, a Sanofi Company, Alnylam Pharmaceuticals, Novo Nordisk, ApcinteX Ltd, Freeline Therapeutics, Sangamo Therapeutics, among others. Hemophilia B Market Hemophilia B Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of market trends, market drivers, market barriers, and key hemophilia B companies such as Gem Pharmaceuticals, Lytix Biopharma, Incyte Corporation, Daiichi Sankyo, NantPharma, Iovance Biotherapeutics, Agenus, Eli Lilly and Company, Adaptimmune, Aadi, AVEO Pharmaceuticals, Amgen, among others. Hemophilia B Pipeline Hemophilia B Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key hemophilia B companies, including Shanghai Vitalgen BioPharma, Belief BioMed, TiumBio, Jiangsu Gensciences, Centessa Pharmaceuticals, Takeda, Sanofi, GC Biopharma Corp, Equilibra Bioscience LLC, Regeneron Pharmaceuticals, ISU Abxis, 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

Research marks one of the longest gene therapy study follow-ups to date PHILADELPHIA, April 17, 2025 /PRNewswire/ -- Researchers at Children's Hospital of Philadelphia (CHOP) in collaboration with researchers at Royal Prince Alfred Hospital in Sydney, Australia announced the results of the longest follow-up study to date for individuals receiving the gene therapy fidanacogene elaparvovec to treat hemophilia B, a rare, X-linked disorder resulting from a deficiency in coagulation factor IX (FIX) activity. The findings, which support the potential of this gene therapy as a safe and effective long-term solution for hemophilia B, were published today in the New England Journal of Medicine. Hemophilia B patients have an inherited gene mutation that impairs their ability to produce normal levels of the blood clotting factor IX, resulting in disabling or life-threatening bleeding that can arise spontaneously or be caused by trauma. The current standard of care for hemophilia B is intravenous factor replacement to prevent or manage bleeding episodes, while simultaneously helping to reduce joint damage and other complications caused by frequent bleeding. However, recombinant adeno-associated virus (rAAV) vectors present the opportunity for a one-time disease-altering therapy. Fidanacogene elaparvovec incorporates a hepatotropic AAV capsid and a high-activity FIX transgene encoding FIX-R338L (also known as FIX-Padua). FIX-Padua, a small natural change in the FIX protein, makes it 8-12 times more effective. Consequently, patients need a much lower dose of the gene therapy to get the right level of FIX activity making this approach a more effective option for treating hemophilia B. In 2017, Lindsey George, MD, a CHOP hematologist with expertise in gene therapy and Director of Clinical In Vivo Gene Therapy at CHOP, led a team on the first successful hemophilia B Padua trial. Published in the New England Journal of Medicine, the researchers described therapeutic expression of factor IX after gene transfer in 10 participants with hemophilia B with one year of follow up. This new study is a long-term progression of that initial research. "Our findings mark the longest-ever follow-up for patients with hemophilia B who received gene therapy with FIX-Padua," said Benjamin J. Samelson-Jones, MD, PhD, a lead study author and an attending physician in the Division of Hematology at CHOP. "These results offer hope that gene therapy for hemophilia B has the potential to transform the standard of care, offering a future with greater independence and improved quality of life for hemophilia patients." In the study, Samelson-Jones and his team, along with Professor John Rasko and his team at RPA Hospital, followed 14 male participants, 18 and older, for a period of 3 to 6 years with 8 participants ongoing. Long-term efficacy was achieved at the lowest intravenous dose of rAAV for any indication. Most patients who received the treatment maintained sustained levels of FIX activity and experienced fewer bleeding episodes, reduced join pain and an overall improvement in physical mobility. Safety data is also encouraging, with no major adverse events linked directly to the therapy. Some patients experienced mild, transient elevations in liver enzyme levels, which were effectively managed with medication. However, post-treatment data suggests that many participants can significantly decrease or even eliminate their reliance on FIX infusions, reducing the burden of treatment and healthcare costs. This work was supported by Pfizer. Rasko and Samelson-Jones, et. al. "Fidanacogene Elaparvovec for Hemophilia B: A Multi-Year Follow-Up Study." NEJM. Online April 17, 2025. DOI: 10.1056/NEJMoa2307159 About Children's Hospital of Philadelphia: A non-profit, charitable organization, Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, the hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. The institution has a well-established history of providing advanced pediatric care close to home through its CHOP Care Network, which includes more than 50 primary care practices, specialty care and surgical centers, urgent care centers, and community hospital alliances throughout Pennsylvania and New Jersey. CHOP also operates the Middleman Family Pavilion and its dedicated pediatric emergency department in King of Prussia, the Behavioral Health and Crisis Center (including a 24/7 Crisis Response Center) and the Center for Advanced Behavioral Healthcare, a mental health outpatient facility. Its unique family-centered care and public service programs have brought Children's Hospital of Philadelphia recognition as a leading advocate for children and adolescents. For more information, visit . Contact: Jennifer Lee Children's Hospital of Philadelphia (267) 426-6084 [email protected] SOURCE Children's Hospital of Philadelphia WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED