Pfizer PFE announced that the FDA has approved its subcutaneously administered drug marstacimab for treating certain hemophilia patients. The drug will be marketed under the trade name Hympavzi.

With this nod, the once-weekly Hympavzi is 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).

The FDA’s approval is based on data from the phase III BASIS study, which evaluated marstacimab in hemophilia patients over a 12-month period. The study achieved its primary endpoint — treatment with marstacimab achieved statistically significant and clinically relevant reduction in annualized bleeding rate (ABR) compared with the current standard-of-care treatment for hemophilia patients.

Following this approval, Hympavzi became the first hemophilia medicine approved in the United States to be administered via a pre-filled, auto-injector pen. A regulatory filing is currently under review in the EU seeking approval for the Hympavziin this indication, also supported by data from the BASIS study.

A rare genetic blood disorder, hemophilia 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.

PFE Stock Performance

Year to date, shares of Pfizer have risen 1.2% compared with the industry’s 21.3% growth.

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Second Hemophilia Nod for Pfizer This Year

The latest FDA decision marks the second approval for Pfizer this year in the hemophilia space. Earlier in April, the FDA approved Beqvez (fidanacogene elaparvovec), the company’s one-shot gene therapy for hemophilia B. Recently, this gene therapy also received approval for a similar use in the EU, where it is marketed under the brand name Durveqtix.

Apart from Beqvez and Hympavzi, Pfizer is also developing an investigational one-time gene therapy for hemophilia A called giroctocogene fitelparvovec. Earlier this 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.

Pfizer’s Competition in the Hemophilia Space

Like Hympavzi, Novo Nordisk NVO also developed its anti-TFPI inhibitor, concizumab, for treating hemophilia A and B with inhibitors. Last year, NVO received a complete response letter (CRL) from the FDA on the regulatory filing for concizumab in hemophilia A or B patients. Per the CRL, the agency requested additional data from Novo Nordisk on the drug’s administration and manufacturing process. The Novo drug was approved for use in Canada and Japan last year, where it is marketed under the brand name Alhemo.

Beqvez faces direct competition from Hemgenix, which was approved by the FDA in 2022 as the first gene therapy for treating hemophilia B in adults aged 18 years and older. This gene therapy has been developed by uniQure QURE, in partnership with CSL Behring, a subsidiary of CSL Limited. The clinical development program for Hemgenix was initially led by uniQure, which was subsequently transferred to CSL after it acquired global rights to commercialize the treatment.

BioMarin Pharmaceuticals’ BMRN Roctavian (valoctocogene roxaparvovec, or valrox) is the first and only gene therapy for hemophilia A, which was approved by the FDA last year after a series of delays and rejections. The BioMarin therapy was initially approved in the EU in August 2022.

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