Strong top-line findings from the Phase 3 BASIS study of Pfizer Inc.’s (NYSE: PFE) experimental monoclonal antibody HYMPAVZITM (marstacimab), a novel inhibitor-based therapy for hemophilia A or B, were announced. In comparison to on-demand therapies, the trial demonstrated that once-weekly subcutaneous medication significantly reduced bleeding rates in adults and adolescents.
The open-label, worldwide, multicenter BASIS trial met its primary objective when patients treated with HYMPAVZI showed a 93% improvement in annualized bleeding rate (ABR) over their previous on-demand intravenous regimen at a 12-month follow-up. Patients on HYMPAVZI had an ABR of 1.39, which was considerably lower than the 19.78 ABR from earlier on-demand treatment.
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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.
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.
Michael Vincent
An uncommon genetic condition called hemophilia impairs the body’s ability to make blood clots, which is necessary to stop bleeding. Patients who take inhibitors develop antibodies, which renders conventional factor replacement therapy useless. Inhibitors increase the risk of severe and recurring bleeding in patients.
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HYMPAVZI is a tissue factor pathway inhibitor (TFPI) that works by restoring equilibrium to the body’s coagulation system rather than substituting the clotting components that are lacking. It is administered using a pre-filled pen, making it a less complicated alternative to intravenous treatment.
In order to obtain approval for HYMPAVZI to treat hemophilia A or B patients with inhibitors, Pfizer plans to communicate these findings to regulatory agencies. At a subsequent medical conference, the complete basic study dataset will be showcased.
Regulatory clearances for HYMPAVZI have been granted in the United States and Europe for eligible patients with hemophilia A who are not taking factor VIII inhibitors or hemophilia B who are not taking factor IX inhibitors. In the United States and the European Union, HYMPAVZI was the first anti-TFPI licensed for the treatment of hemophilia A or B. It was also the first hemophilia medication authorized for use with a pre-filled, auto-injector pen. For those who qualify, it is the first once-weekly subcutaneous preventive therapy for hemophilia B. HYMPAVZI can provide a once-weekly subcutaneous therapy alternative that requires little preparation for each medication administration.
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