Key Takeaways
- HYMPAVZI™, Pfizer’s new once-weekly subcutaneous treatment, drastically reduces bleeding episodes in hemophilia A and B patients without inhibitors.
- The FDA-approved therapy simplifies treatment with a pre-filled pen, replacing frequent IV infusions for eligible patients aged 12 and older.
- Clinical trials show a 92% reduction in bleeding for hemophilia B and 35% for hemophilia A, marking a major breakthrough in hemophilia care.
Pfizer has achieved a major milestone with the FDA approval of HYMPAVZI™ (marstacimab-hncq), a groundbreaking treatment that offers once-weekly prophylaxis for people living with hemophilia A or B without inhibitors. This approval signals a new era in hemophilia care, offering a more convenient and effective alternative to traditional treatment approaches that often involve multiple intravenous infusions per week.
For the first time in the U.S., patients can access a pre-filled pen or syringe for hemophilia B treatment, making administration significantly easier. HYMPAVZI™ also marks a significant advancement for hemophilia A, offering improved bleed prevention with minimal preparation. The approval is based on Pfizer’s pivotal Phase 3 BASIS trial, which demonstrated substantial reductions in annualized bleeding rates compared to traditional treatments. “HYMPAVZI is a meaningful advancement for people living with hemophilia A or B without inhibitors for bleed prevention,” said Dr. Suchitra Acharya, Director of the Hemostasis and Thrombosis Center at Northwell Health.
“With once-weekly dosing and a generally manageable safety profile, this treatment reduces the time-consuming and cumbersome nature of traditional therapy.” Hemophilia, a rare genetic disorder that affects over 800,000 people worldwide, inhibits the body’s ability to form blood clots properly, often resulting in painful and potentially dangerous bleeding episodes. Current treatment options, especially for those with severe hemophilia, often require frequent IV infusions multiple times a week. These infusions can be challenging to maintain, with adherence rates low due to the time-consuming nature of treatment.
HYMPAVZI™ seeks to change the game by providing a simple, once-weekly subcutaneous injection that significantly reduces bleeding episodes. For those living with hemophilia B, it becomes the first U.S.-approved subcutaneous prophylactic treatment, replacing the need for frequent intravenous infusions. “HYMPAVZI brings a new standard of care for hemophilia, simplifying treatment while improving outcomes for patients,” said Aamir Malik, Chief U.S. Commercial Officer at Pfizer. “It is our second hemophilia treatment approved this year, showcasing our commitment to advancing care for these patients.”
Transformative Results from Phase 3 Clinical Trials
The approval of HYMPAVZI™ is backed by Phase 3 BASIS trial results, which assessed the drug’s efficacy and safety in adolescents and adults with hemophilia A and B without inhibitors. The study included 116 participants who received weekly HYMPAVZI injections.
Patients with hemophilia A experienced a 35% reduction in annualized bleeding rates compared to those receiving routine prophylaxis. Patients with hemophilia B saw a 92% reduction in bleeding episodes compared to on-demand treatment.The treatment also proved non-inferior to routine prophylaxis in hemophilia A for bleeding-related secondary outcomes, such as joint and spontaneous bleeds.
The most commonly reported side effects included injection site reactions, headache, and itching, all of which were consistent with earlier trial data. Importantly, HYMPAVZI™ proved to be well-tolerated with a favorable safety profile, positioning it as a promising option for hemophilia patients seeking both convenience and efficacy.
Implications for Hemophilia Community
With this approval, Pfizer now offers a diverse portfolio of hemophilia treatments, ranging from gene therapies to recombinant factor treatments. HYMPAVZI™ joins the ranks of groundbreaking therapies, providing patients and healthcare providers with more options to tailor treatment plans according to individual needs.
“HYMPAVZI represents a powerful step forward in advancing care for the hemophilia community,” said Phil Gattone, President of the National Bleeding Disorders Foundation. “For many, this therapy will dramatically simplify daily life while offering better bleed prevention.”
Hemophilia has historically been a difficult condition to manage, with bleeding episodes resulting in chronic joint damage and diminished quality of life. HYMPAVZI™ offers hope for better treatment adherence and long-term outcomes, particularly for those patients for whom frequent intravenous infusions were previously the only option.
Pfizer’s efforts to combat hemophilia are part of a broader mission to innovate and expand treatment options for rare diseases. With HYMPAVZI™, patients now have access to an advanced treatment with the potential to significantly improve their quality of life. The approval follows the recent regulatory successes of Pfizer’s hemophilia B gene therapy BEQVEZ™, and positive results from its hemophilia A gene therapy program. The BASIS study continues with ongoing trials, including investigations into HYMPAVZI™’s effects on children and patients with inhibitors, with results expected in 2025.
Resource: Pfizer, October 11, 2024
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