Pfizer Discontinues Hemophilia Gene Therapy Beqvez As Interest Wanes

Pfizer, a leading pharmaceutical company, has announced its decision to discontinue the development and commercialization of its hemophilia B gene therapy, Beqvez (fidanacogene elaparvovec). The move comes less than a year after the therapy's approval by the U.S. Food and Drug Administration (FDA) and reflects the limited interest from patients and healthcare providers in hemophilia gene therapies to date.

Beqvez, a one-time treatment with a list price of $3.5 million per dose, was approved in April 2024 for the treatment of adult men with moderate to severe hemophilia B who have a history of life-threatening hemorrhage or repeated serious spontaneous bleeding episodes. However, no patients seem to have received commercial Beqvez since its approval, indicating a lack of demand for the therapy.

Pfizer's decision to discontinue Beqvez is part of a broader trend in the hemophilia gene therapy market. Other companies, such as CSL and BioMarin, have also faced challenges in launching and commercializing their gene therapies for hemophilia. CSL's Hemgenix, the first-to-market rival for hemophilia B, has faced a slower-than-expected launch due to a fragmented U.S. healthcare system. Similarly, BioMarin's Roctavian, a gene therapy for hemophilia A, has had a slow sales ramp and limited commercial focus.

The high cost of gene therapies, such as Beqvez, Hemgenix, and Roctavian, may pose reimbursement challenges for payers and healthcare systems. Additionally, the fragmented U.S. healthcare system can make it difficult for advanced therapies like gene therapies to be adopted. The competition from existing treatments, such as Roche's Hemlibra, which booked 4.5 billion Swiss francs ($5 billion) in global revenue in 2024, may also contribute to the limited interest in hemophilia gene therapies.

Pfizer's decision to discontinue Beqvez has significant implications for the company's overall gene therapy pipeline and strategy. Following the termination of the Sangamo partnership and the discontinuation of Beqvez, Pfizer is shifting its hemophilia resources to Hympavzi, a non-factor treatment option for hemophilia A and B. This indicates a strategic pivot away from gene therapies and towards other treatment modalities in the hemophilia space.

The discontinuation of Beqvez also has potential implications for the broader hemophilia gene therapy market and other competitors, such as CSL and BioMarin. The limited competition may lead to increased market share for CSL and BioMarin, as they will have fewer alternatives for patients and healthcare providers to consider. However, the slow uptake of Hemgenix and Roctavian, as well as Pfizer's decision to discontinue Beqvez, suggests that there may be underlying challenges in the hemophilia gene therapy market that other competitors should be aware of.

In conclusion, Pfizer's decision to discontinue Beqvez reflects the limited interest in hemophilia gene therapies and has significant implications for the company's gene therapy pipeline and strategy. The broader hemophilia gene therapy market and other competitors, such as CSL and BioMarin, should be aware of the challenges and potential opportunities presented by this development.