As Pfizer backs out of hemophilia gene therapy space, CSL hopes Hemgenix is here to stay

Title

Pfizer Exits Hemophilia Gene Therapy Space, CSL Stakes Future on Hemgenix

Keywords

Pfizer hemophilia gene therapy exit
Hemgenix gene therapy
Hemophilia B treatment advancements
Beqvez discontinuation
CSL Behring Hemgenix success
Gene therapy challenges
Factor IX gene therapy

Key Facts

- Pfizer's Exit from Gene Therapy:
Pfizer has discontinued its hemophilia B gene therapy, Beqvez, citing weak demand and limited adoption among doctors and patients despite its FDA approval in April 2024. This decision marks Pfizer’s full withdrawal from the gene therapy field for hemophilia, as it focuses on alternatives like Hympavzi, a subcutaneous antibody treatment for hemophilia A and B1 4 9.

- Beqvez's Challenges:
Beqvez carried a high price tag of $3.5 million per dose but failed to secure any commercial patients outside of clinical trials, signaling a lack of market traction. The limited interest may stem from competition, pricing concerns, and availability of emerging alternatives like CSL Behring’s Hemgenix1 4 9.

- CSL’s Commitment to Hemgenix:
Hemgenix, the first FDA-approved gene therapy for hemophilia B, offers a one-time infusion that sustains elevated factor IX levels and reduces annualized bleeding rates by 90% over four years, as seen in clinical trials. The therapy eliminates the need for regular prophylactic infusions for the majority of patients2 3 7.

- Unique Features of Hemgenix:
Unlike its competitors, Hemgenix can treat patients with or without pre-existing neutralizing antibodies to adeno-associated virus type 5 (AAV5). It is also associated with sustained efficacy lasting up to four years in clinical trials, with patients achieving independence from routine factor IX prophylaxis2 3 8.

- Market and Adoption Challenges:
Despite its promising results, Hemgenix faces hurdles in uptake due to its high cost of $3.5 million per treatment and limited reimbursement frameworks. The fragmented healthcare system in regions like the U.S. has also slowed adoption1 8 9.

- Evolving Landscape of Hemophilia Treatments:
The discontinuation of Beqvez leaves CSL’s Hemgenix as a leading option for hemophilia B gene therapy. Meanwhile, emerging alternatives such as subcutaneous treatments like Hympavzi and rebalancing agents like Sanofi’s fitusiran offer innovative options that could redefine disease management4 6 9.

- A Shift in Focus:
Pfizer and other pharmaceutical companies may shift resources away from first-generation gene therapies to explore more cost-effective and patient-friendly treatments in areas like mRNA technologies and rebalancing mechanisms1 9.