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.

PFE Stock Performance

Year to date, shares of Pfizer have lost 13.4% against the industry’s 3.1% growth.

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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.

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. Last month, the EMA’s CHMP recommended approving Novo’s drug to treat patients aged 12 years or older living with hemophilia A or B with inhibitors. The NVO drug is already approved for use in Canada and Japan since 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 was 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.

Appointment of the New Chief Scientific Officer and R&D President

In a separate press release, Pfizer announced that it has appointed Dr. Chris Boshoff as the chief scientific officer and president, Research & Development. He will succeed Dr. Mikael Dolsten, whose departure was announced earlier this year.

This new appointment will be effective from Jan. 1, 2025. Dr. Boshoff currently serves as the chief oncology officer and executive vice president of the company.

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