 NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) today announced that the U.S. Food and Drug Administration (FDA) has approved an expanded indication for HYMPAVZI® (marstacimab-hncq) to include the treatment of patients with hemophilia A or B 12 years and older with inhibitors and pediatric patients (ages 6 to 11 years) with or without inhibitors. HYMPAVZI is now indicated in the U.S. for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients 6 years of age and older with hemophilia A (congenital factor VIII deficiency) with or without factor VIII inhibitors, or hemophilia B (congenital factor IX deficiency) with or without factor IX inhibitors. HYMPAVZI offers a combination of prophylactic bleed protection with a straightforward, once-weekly subcutaneous administration that does not require routine treatment-related lab monitoring. “For children who have to deal with bleeding episodes from an early age and for people living with hemophilia who develop inhibitors, treatment options have been limited and are often burdensome,” said Guy Young, M.D., Director, Hemostasis and Thrombosis Center at Children's Hospital, Los Angeles. “A treatment that can reduce bleeding with straightforward, once-weekly administration has the potential to fundamentally change how patients and caregivers approach this disease, offering control with a level of simplicity this community has long needed.” Hemophilia is typically diagnosed in early childhood and impacts more than 800,000 people worldwide. The inability of the blood to clot properly can increase the risk of painful bleeding, including inside the joints, which can cause joint scarring and damage. Children’s joints have growing cartilage and bone, which makes them particularly susceptible to damage caused by repeated bleeding episodes. Inhibitors to factor replacement therapy limit treatment options for people living with hemophilia and are associated with an increased risk of uncontrolled bleeding. These inhibitory antibodies develop in approximately 20% of those with hemophilia A and 3% of those with hemophilia B. Many people living with inhibitors to FVIII and FIX are unable to continue taking factor replacement therapies as they no longer prevent or stop bleeding episodes, particularly in individuals who are refractory to immune tolerance induction therapy. “With this expanded approval, we believe HYMPAVZI can become a transformative option and meet a significant medical need for people living with hemophilia A or B with or without inhibitors ages 6 years and older. Particularly for children ages 6 to 11 with hemophilia B who will now, for the first time, have a subcutaneous non-factor treatment available,” said Aamir Malik, Chief U.S. Commercial Officer and Executive Vice President, Pfizer. “This milestone represents the latest step in Pfizer’s more than 40-year commitment to advancing care and quality of life for people living with hemophilia, which began with the introduction of recombinant therapies and has evolved with the introduction of this once-weekly subcutaneous treatment.” Results from the Phase 3 BASIS trial (NCT03938792) supported the approval of HYMPAVZI in adults and adolescents 12 years and older with hemophilia A or B with inhibitors. The results demonstrated the superiority of HYMPAVZI in improving key bleeding outcomes including significantly reducing mean treated annualized bleeding rate (ABR) by 93% compared to on-demand (OD) intravenous treatment with bypassing agents (1.4 [95% CI: 0.9-2.3] vs.19.8 [95% CI: 16.1-24.3]; p<0.0001). Interim results from the Phase 3 BASIS KIDS trial (NCT05611801) supported the approval of HYMPAVZI in children ages 6 to 17 years with hemophilia A or B with or without inhibitors. Descriptive analyses, which summarize trends in the data, in patients who received HYMPAVZI demonstrated:
The most commonly reported adverse reactions (≥2%) in adult and pediatric patients 6 years of age and older with or without inhibitors were injection site reaction, headache, pyrexia, arthralgia, diarrhea, pruritus, and rash. Thromboembolic events (venous and arterial) in two patients were observed among a total of 259 patients who received HYMPAVZI in the open-label extension study. Thromboembolic events, hypersensitivity, embryofetal toxicity, and increased laboratory values of fibrin D-dimer and prothrombin fragment 1.2 are noted within the Warnings and Precautions section of the U.S. label. This HYMPAVZI application was reviewed under FDA Priority Review, which is granted to medicines that treat a serious condition and provide a significant improvement in safety or effectiveness over available therapy. The FDA also granted HYMPAVZI Breakthrough Therapy Designation for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in younger pediatric (≥6 to <12 years of age) patients with hemophilia B with and without inhibitors. The FDA’s Breakthrough Therapy Designation is intended to expedite the development and review of medicines with the potential to treat a serious or life-threatening disease when preliminary clinical evidence indicates the medicine may demonstrate substantial improvement on a clinically significant endpoint over available therapies. Read More: Pfizer Inc. - U.S. FDA Approves Pfizer’s HYMPAVZI for the Treatment of Two Additional Hemophilia A or B Patient Populations with Significant Medical Need
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 Dear Hemophilia B Community,
We are writing to share an important update on the availability of HEMGENIX® (etranacogene dezaparvovec-drlb). CSL is currently experiencing a temporary global stockout of HEMGENIX® that will result in delays in treatment for some individuals in countries with established commercial access. We want to be very clear that this situation is not related to the safety or effectiveness of HEMGENIX®. Rather, it reflects the complexity of manufacturing gene therapies, and our commitment to adhering to the highest regulatory and quality standards for the people we serve. We are working with regulatory authorities on strategies to ensure stable ongoing supply for HEMGENIX® while preserving our high-quality standards. We know that this update may raise questions and that’s completely understandable. What we want you to know is that our highest priority, as always, is ensuring that eligible individuals have the information they need to determine if HEMGENIX® is right for them, and that when they do, HEMGENIX® is available in accordance with the highest standards. We remain fully committed to delivering this innovative, one-time gene therapy to the hemophilia B community and continue to have strong confidence in HEMGENIX®. We understand the careful thought and planning that go into every individual decision about gene therapy treatment. We deeply value our long‑standing partnership of trust with the hemophilia B community and will continue to keep you updated to ensure people with hemophilia B and their healthcare providers are informed and supported as they plan their hemophilia care needs. For further information, please visit our contact page on CSL.com here: https://www.csl.com/contact. Sincerely, Dr. Deborah Long, SVP, Medical Affairs Because Women’s Bleeding Experiences Matter: MicrohealthDigital Hematology Launches the PULSE Pilot3/3/2026  Microhealth, the leading global app in digital hematology, is launching a new digital health
initiative, the PULSE (Period Understanding and Logging of Symptoms for Empowerment) Pilot in the United States to address a critical gap in bleeding disorders care: individuals are undertreated due to the normalization of menstrual bleeding, leading to gaps in access to appropriate treatment and care. The goal of the PULSE Pilot is to inform a potential expansion of the existing bleed log within the Microhealth Bleeding Disorders app, specifically to capture menstrual bleeding and related symptoms more effectively, as well as patient-reported outcomes. PULSE Pilot volunteers will securely log menstrual cycles and related symptoms with active consent. Microhealth will collect data in alignment with company data practices, de-identify and aggregate it, and use it to inform future app development and quality improvement. This is not a clinical research study. Enrollment is available for individuals in the United States who menstruate and have a confirmed bleeding disorder diagnosis or are suspected of having a bleeding disorder. Volunteers can participate regardless of current treatment or medication status. For over 15 years, Microhealth Digital Hematology has been an internationally trusted, free digital tool for the bleeding disorders community worldwide. It lets individuals track bleeds, symptoms, treatments, physical activity, and health experiences in one place; empowering them to own their data, advocate for themselves, and optionally connect with Hemophilia Treatment Centers. The Microhealth Bleeding Disorders app provides care teams with free, real-time, patient-reported data that reflects their individual lived experience. With the launch of the PULSE Pilot, Microhealth Digital Hematology aims to make individuals' menstrual experiences more recognized and valued. Interested in volunteering for PULSE? Please scan the QR code or visit https://forms.gle/p8eu9FRV8XZFkMDP6 to join this critical effort. For further information, please contact Microhealth Community Digital Advocates Nandini Pethe at [email protected] or Michelle Cecil at [email protected]. For more information about Microhealth, visit Microhealth.com.  We are pleased to announce an important update to the IDELVION Connect Copay Program.* In recognition of the ongoing needs of the patient community and our commitment to providing meaningful support, we are increasing the maximum eligible out-of-pocket copay assistance from $12,000 to $20,000 per patient. To enroll, patients and Specialty Pharmacies are encouraged to contact IDELVION Connect at 1-800-676-4266.
What is Changing
What is Not Changing
If you have questions, feel free to contact IDELVION Connect at 1-800-676-4266.  Dear Hemophilia Community Partners,
On December 22, 2025, we shared the very sad news of the passing of a hemophilia A participant with inhibitors in the marstacimab long-term extension trial. We remain committed to transparency as we continue to assess what occurred. The circumstances of this case are multi-factorial – including co-existing conditions, the participant undergoing a urethroscopic surgical procedure, and use of a bypassing agent as a concomitant therapy – and we are working to understand these elements as fully as possible in this clinical setting. We plan to present this information as part of a late-breaker session at the EAHAD congress on February 6. As we previously shared, an independent external Data Monitoring Committee oversees all ongoing marstacimab studies. Based on the current information available, the studies are continuing as planned and the established benefit/risk profile of marstacimab remains unchanged. We are continuing to review and assess information on this case as we always do for our medicines, per established processes. Thank you for your partnership and for your commitment to the hemophilia community. Sincerely, The Pfizer Hemophilia Team  Dear Hemophilia Community Partners,
We are deeply saddened to share that a participant in the long-term extension trial (B7841007) studying marstacimab in people living with hemophilia A or B with or without inhibitors has passed away in December 2025 following serious adverse events of cerebellar infarction and subsequent cerebral hemorrhage. On behalf of everyone at Pfizer, we extend our heartfelt sympathies to the participant’s family, friends, and all those involved in their care. Pfizer, together with the trial investigator and the independent external Data Monitoring Committee, are actively gathering information to better understand the complex, multi factorial circumstances surrounding this occurrence – including co-existing medical conditions and concomitant medications – and the causality of the event. The individual, a patient with hemophilia A and inhibitors, participated in the active treatment phase of the parent study (B7841005) in 2022 prior to entering the long-term extension study in 2023. Regulatory authorities and investigators have been informed. The safety and well-being of participants in our clinical trials remain our highest priority, and we are committed to transparency and keeping the community informed as we learn more. Sincerely, The Pfizer Hemophilia Team Pfizer Announces Positive Topline Phase 3 Results for HYMPAVZI™ in Hemophilia A or B with Inhibitors7/22/2025  NEW YORK, June 26, 2025
Pfizer Inc. (NYSE: PFE) today announced positive topline results from the Phase 3 BASIS study (NCT03938792) evaluating HYMPAVZI™ (marstacimab) for adults and adolescents living with hemophilia A or B with inhibitors. The study met the primary endpoint and key secondary bleeding endpoints demonstrating the superiority of once-weekly subcutaneous HYMPAVZI in improving key bleeding outcomes compared to on- demand treatment in a patient population where less burdensome treatment approaches are needed. Inhibitors, or antibodies, which neutralize factor replacement therapies and render them ineffective, may develop in people living with hemophilia. Inhibitors can be diagnosed with a blood test. Of the more than 800,000 people in the world living with hemophilia A or hemophilia B, approximately 20% of people with hemophilia A and 3% of people with hemophilia B are unable to continue taking factor replacement therapies because they develop inhibitors to FVIII (Factor VIII) and FIX (Factor IX) and these therapies no longer prevent or stop bleeding episodes. “Patients with inhibitors tend to face frequent complications, and navigating the treatment landscape can introduce complexities and increase disease burden,” said Davide Matino, M.D., M.Sc., BASIS Principal Investigator, Associate Professor of Medicine, McMaster University. “The strong bleed reduction with HYMPAVZI compared to on-demand treatment in the Phase 3 BASIS study, coupled with its weekly administration method, offers exciting potential for these patients who are in critical need of treatment options.” The BASIS trial demonstrated that prophylactic treatment with HYMPAVZI resulted in a statistically significant and clinically relevant reduction in annualized bleeding rate (ABR) of treated bleeds in people living with severe hemophilia A or hemophilia B with inhibitors. Forty-eight people living with hemophilia were treated with HYMPAVZI during a 12-month period versus an on-demand intravenous regimen with bypassing agents, administered as part of usual care in the six-month lead-in period. HYMPAVZI was superior to on-demand treatment with a 93% reduction in ABR over 12 months (ABR 1.39 vs ABR on-demand 19.78; p < 0.0001). Superiority of HYMPAVZI was also demonstrated across all bleeding-related secondary endpoints—spontaneous bleeds, joint bleeds, target joint bleeds, and total bleeds. HYMPAVZI was generally well-tolerated, consistent with the non-inhibitor cohort of the BASIS study and Phase 1/2 results. No deaths or thromboembolic events were reported. “These encouraging results demonstrate HYMPAVZI’s potential to help people living with hemophilia A or B with inhibitors, meeting an important need for patients with antibodies that neutralize most factor-based prophylactic options used to manage bleeding episodes,” said Michael Vincent, M.D., Ph.D., Chief Inflammation & Immunology Officer, Pfizer. “HYMPAVZI represents Pfizer’s latest contribution in more than 40 years of working to advance hemophilia care, as a generally well-tolerated treatment option that could offer bleed protection with a straightforward, once-weekly subcutaneous administration in a pre-filled pen for patients with inhibitors, if approved in this patient population.” Analyses of the full Phase 3 dataset from the inhibitor cohort of the BASIS study are ongoing, and additional data will be presented at upcoming medical meetings. Pfizer plans to discuss these data with regulatory authorities, with the goal of initiating regulatory filings for HYMPAVZI for the treatment of patients living with hemophilia with inhibitors. Discovered by Pfizer scientists, HYMPAVZI has a mechanism of action that is differentiated from FVIII and FIX replacement treatments. Instead of replacing missing or insufficient clotting factors, HYMPAVZI is intentionally designed to target tissue factor pathway inhibitor (TFPI), one of the body’s natural mechanisms that inhibits the initiation of blood clotting. By targeting the Kunitz 2 domain of TFPI, HYMPAVZI may help re-establish balance between bleeding and blood clot formation with the goal of offering a combination of bleed protection, good tolerability, and straightforward administration. Read more: https://www.pfizer.com/news/press-release/press-release-detail/pfizer-announces-positive-topline-phase-3-results
May 27, 2025
Dear members of the hemophilia community, We are reaching out to share an important update about the integration of Spark Therapeutics into the Roche Group and to reiterate the commitment to develop a gene therapy as well as other therapeutic advances for people with hemophilia. In 2019, Spark Therapeutics became a member of the Roche Group. With its extensive resources and worldwide reach, including Genentech in the United States, this partnership aimed to accelerate the discovery, development and delivery of potential gene therapies for more patients affected by a wide range of genetic diseases, including but not limited to hemophilia. On January 30, 2025, Roche, in support of its commitment to address unmet medical needs through cutting-edge science, announced that Spark will become fully integrated into the Roche Group. As a result, in the U.S. Genentech will be the point of contact for the hemophilia A gene therapy program going forward. While this means that Spark Therapeutics will no longer operate as an independent company, its expertise and knowledge as a leader in gene therapy will be folded into Roche and Genentech, and it will continue in research and manufacturing of medicines. This integration of Spark into the Roche Group supports the long-standing ambition and commitment to provide multiple transformative and reliable treatment options that can address the needs of all people with hemophilia A, their families, and caregivers. With this change, we want to reinforce that the Roche Group remains committed to discovering, developing and commercializing innovative treatments for hemophilia. In December 2024, we announced the decision to introduce an enhanced function factor VIII variant (SPK-8011QQ) into the hemophilia A gene therapy program. This next-generation gene therapy program is planned to be studied in a Phase 2b clinical trial sponsored by Roche and Genentech. More detailed information about the program is available at the end of this letter. For those who participated in the Phase 1/2 clinical trial of investigational dirloctocogene samoparvovec (SPK-8011), your participation has been invaluable to advancing hemophilia A gene therapy. The long- term follow-up study will continue, and you will continue to be followed as a study participant. We encourage you to reach out to your clinical trial site with any questions you may have. Our team wants to take a moment to express our deepest gratitude to you–the patients, families, caregivers, advocacy group leaders, and healthcare providers. Over the years, you have shared your experiences living with hemophilia, we have heard your hopes, questions and curiosities about gene therapy, and you have actively engaged in clinical research–all of which has been essential in advancing gene therapy for hemophilia forward. Going forward, please direct any questions about the program or Spark integration, to Genentech at [email protected]. We look forward to continuing this close collaboration through the Genentech Patient Advocacy Relations team. Sincerely, Tessa Field, Spark Therapeutics, Director, Patient Advocacy Gina Truslow, Genentech, Director, Patient Advocacy Relations Phase 2b Hemophilia A Gene Therapy Clinical Trial In line with the commitment to bring transformational therapies to patients, the Roche Group is introducing an enhanced function factor VIII variant (SPK-8011QQ) into the hemophilia A gene therapy program. Current adeno-associated virus (AAV) gene therapies introduce the factor VIII gene to the liver aiming to improve blood clotting for individuals with hemophilia A. By modifying the gene used in our investigational gene therapy, we hope to achieve what the hemophilia community and healthcare providers are looking for in one-time gene therapies, including improved hemostasis and lowered treatment burden. This decision builds on the results seen in the phase 1/2 study of investigational gene therapy dirloctocogene samoparvovec (SPK-8011), which provided early insights to safety, predictability, and durability using a low-dose approach. This study is investigational and efficacy and safety of dirloctocogene samoparvovec has not been established. We will be leveraging the capsid (delivery vehicle) and scientific learnings from the phase 1/2 program and introducing an enhanced function factor VIII variant with the goal of developing a durable gene therapy that provides effective protection against bleeds, without the need for factor VIII prophylaxis. As we transition to the enhanced function FVIII variant, we have chosen to discontinue the phase 3 dirloctocogene samoparvovec gene therapy study. The phase 3 was stopped prior to dosing any participants. Stopping the study was not related to safety concerns with dirloctocogene samoparvovec. Participants in the phase 1/2 study of dirloctocogene samoparvovec will continue to receive long-term follow-up and monitoring. We are excited about the potential to advance hemophilia A gene therapy through a next-generation program. This next generation gene therapy program is planned to be studied in a Phase 2b clinical trial sponsored by Roche and Genentech. The phase 2b study will allow us an opportunity to gather safety data before starting a larger phase 3 study of the enhanced function variant.  Qfitlia approved as the first therapy in the US to treat hemophilia A or B with or without inhibitors
Paris, March 28, 2025. The US Food and Drug Administration (FDA) has approved Qfitlia (fitusiran), the first antithrombin-lowering (AT) therapy for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients (aged 12 or older) with hemophilia A or B with or without factor VIII or IX inhibitors. The approval is based on data from the ATLAS phase 3 studies that demonstrated clinically meaningful bleed protection as measured by annualized bleeding rates (ABR) across hemophilia patients with or without inhibitors. Phil Gattone President and CEO, National Bleeding Disorders Foundation “Current treatment options can make people with hemophilia feel they need to choose between effective bleed control and convenient dosing schedules, leading to trade-offs when it comes to disease management. Qfitlia takes a novel approach to providing protection for people living with hemophilia while reducing the frequency of dosing for patients and their families.” By lowering AT, a protein that inhibits blood clotting, Qfitlia helps increase thrombin generation to restore hemostasis in people with hemophilia. Qfitlia uses small-interfering RNA technology, which enables low treatment frequency, subcutaneous dosing, and low volume injections. Brian Foard Executive Vice President, Head of Specialty Care, Sanofi “This approval highlights our commitment to advancing innovation and improving care for the rare blood disorders community. Qfitlia has the potential to meaningfully change the hemophilia landscape through effective bleed protection, infrequent dosing, and simplified administration. Our robust portfolio of hemophilia treatment options continues to grow as we focus on offering protection with reduced treatment burden that best fits an individual’s needs.” Guy Young, MD Director, Hemostasis and Thrombosis Center at Children's Hospital, Los Angeles “Qfitlia delivers the fewest doses of any prophylactic therapy in hemophilia, and its unique mechanism allows it to be used to treat all types of hemophilia, including with inhibitors and hemophilia B, where unmet medical needs remain. By targeting antithrombin, which can be reliably measured with an FDA-cleared blood assay, Qfitlia is proven to help rebalance hemostasis and improve bleed rates and protection.” In the ATLAS clinical development program, Qfitlia demonstrated low bleed rates across subgroups with as few as six injections a year. Key results include:
In conjunction with the Qfitlia approval, the FDA also cleared the Siemens Healthineers’ INNOVANCE® Antithrombin assay as a companion diagnostic for Qfitlia to measure AT levels. Through the Qfitlia Testing Program with Labcorp, the FDA-cleared companion diagnostic will be available to patients prescribed Qfitlia to measure AT levels at no cost. Qfitlia can offer the fewest doses of all prophylactic therapies, and it will have a comparable price to other prophylactic hemophilia treatments. HemAssist is launching alongside Qfitlia to provide comprehensive patient support services, including insurance and financial assistance as well as educational resources. This program is for patients prescribed Qfitlia or other hemophilia treatments from Sanofi’s portfolio. The FDA granted Qfitlia Orphan Drug Designation for hemophilia A and B, Fast Track Designation for hemophilia A and B with and without factor VIII or IX inhibitors, and Breakthrough Therapy Designation for hemophilia B with factor IX inhibitors. A regulatory submission for Qfitlia for the treatment of hemophilia A or B in adults and adolescents with or without inhibitors is under review in Brazil. A regulatory decision is expected in China in the second half of 2025. Read the full press release: https://www.news.sanofi.us/2025-03-28-Qfitlia-approved-as-the-first-therapy-in-the-US-to-treat-hemophilia-A-or-B-with-or-without-inhibitors  Kedrion Expands Ryplazim Distribution Network for PLGD-1 Patients FORT LEE, NJ., Mar. 20, 2025 /PRNewswire/ -- Kedrion Biopharma Inc. expands the distribution network for Ryplazim, a plasma-derived human plasminogen indicated for treating patients with plasminogen deficiency type 1 (PLGD-1). This expansion ensures timely access to this vital medication for patients across the US. PLGD-1 is a serious condition causing abnormal fibrin-rich lesions on mucosal surfaces, potentially leading to severe consequences like vision and hearing loss, airway obstruction, and infertility. Ryplazim, the only FDA approved therapy for PLGD-1 patients, offers an important treatment option. The recent FDA approval of the technology transfer and manufacturing capacity expansion of Ryplazim has enabled Kedrion to broaden its limited distribution network due to the increase of supply. This crucial step means more patients can have access to this treatment. The expanded network now includes a new distribution partner and two new specialty pharmacies. CuraScript SD a provider of comprehensive distribution solutions, joins existing partners FFF Enterprises and The Alliance Pharmacy in distributing Ryplazim to patients with PLGD-1. CVS Health and Soleo Health have been added to the list of specialty pharmacies carrying Ryplazim, increasing access points for patients. These pharmacies join Nufactor, enhancing the network to ensure wider availability. This collaborative effort underscores Kedrion's commitment to improving patient care and broadening access to essential therapies. To learn more about accessing Ryplazim and its potential benefits, please visit www.Ryplazim.com. Read the full press release: https://lnkd.in/ez242whw 
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