Pfizer Secures Approval for Hemophilia Drug Hympavzi in the EU
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Pfizer PFE announced that the European Commission (“EC”) has granted marketing authorization for Hympavzi (marstacimab) to treat certain hemophilia patients.
With this nod, the once-weekly Hympavzi gets approved for routine prophylaxis (RP) to prevent or reduce the frequency of bleeding episodes in patients aged 12 years and older with hemophilia A or B, without inhibitors to Factor VIII (FVIII) or Factor IX (FIX). Following this approval, Hympavzi became the first hemophilia medicine approved in the United States to be administered via a pre-filled, auto-injector pen.
This approval was expected as the European Medicines Agency's (“EMA”) Committee for Medicinal Products for Human Use (“CHMP”) issued a positive opinion recommending Hympavzi’s approval in the above indication in September.
The EC and CHMP decisions are based on data from the phase III BASIS study, which evaluated Hympavzi in hemophilia patients over a 12-month period. The study achieved its primary endpoint — treatment with the drug achieved statistically significant and clinically relevant reduction in annualized bleeding rate (ABR) compared with the current standard-of-care treatment for hemophilia patients. Based on these results, the FDA approved this drug for a similar indication last month.
Hemophilia, a rare genetic blood disorder, is caused by a missing clotting protein (FVIII in case of hemophilia A and FIX in case of hemophilia B), which prevents normal blood clotting. If the blood does not clot properly, it can lead to painful bleeding inside the joints that can cause scarring and damage. Per Pfizer, Hympavzi targets tissue factor pathway inhibitor (TFPI), which is a natural anticoagulation protein that functions to prevent the formation of blood clots.
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Second Hemophilia Nod for Pfizer This Year in the EU
The latest decision marks the second EC approval for Pfizer this year in the hemophilia space. Earlier in July, the FDA approved Durveqtix (fidanacogene elaparvovec), the company’s one-shot gene therapy for hemophilia B. This gene therapy also received approval for a similar use by the FDA in April, where it is marketed under the brand name Beqvez.
Apart from Durveqtix/Beqvez and Hympavzi, Pfizer is also developing an investigational one-time gene therapy for hemophilia A called giroctocogene fitelparvovec. In July, Pfizer reported positive results from the late-stage AFFINE study, which showed that treatment with the hemophilia A gene therapy led to a statistically significant reduction in ABR compared to the pre-infusion period.