Pfizer Discontinues Hemophilia B Gene Therapy Beqvez, Exits Gene Therapy Field

Pfizer has decided to discontinue global development and commercialization of Beqvez, its FDA-approved hemophilia B gene therapy1 3.

Beqvez was approved by the FDA in April 2024 and had a list price of $3.5 million per dose1 2.

The decision was made due to limited interest from patients and doctors in hemophilia gene therapies1 3.

No patients received commercial Beqvez since its FDA approval1.

With Beqvez's discontinuation, Pfizer currently has no active gene therapy programs in its portfolio1 4.

This move follows Pfizer's recent exit from other gene therapy projects, including:

Termination of a hemophilia A gene therapy partnership with Sangamo Therapeutics1 2
Sale of preclinical gene therapy programs to AstraZeneca's Alexion for up to $1 billion in 20231 10
Abandonment of a Duchenne muscular dystrophy gene therapy program after a phase 3 failure1

Pfizer is shifting its hemophilia resources to Hympavzi, a once-weekly nonfactor antibody drug approved for hemophilia A and B1.

The company's gene therapy manufacturing facility in Sanford, North Carolina, may face potential job cuts related to Beqvez's termination1.

Pfizer's experience mirrors broader struggles in the hemophilia gene therapy field, with other companies like CSL also facing slow adoption of their treatments1.

Despite this setback, Pfizer remains involved in a research collaboration with Beam Therapeutics focused on mRNA-delivered gene editing for rare genetic diseases1.