Pfizer Halts $3.5M Hemophilia B Gene Therapy Beqvez, Exits Gene Therapy Field

Recently, Pfizer announced the cessation of global development and commercialization of Beqvez, a gene therapy for hemophilia B, marking Pfizer's further exit from the gene therapy field.

Termination of Beqvez

Pfizer Decides to Terminate Development and Commercialization of Beqvez Less Than a Year After FDA Approval. Beqvez, a one-time treatment option priced at $3.5 million per patient, faced limited interest from patients and doctors regarding hemophilia gene therapy, leading Pfizer to halt the project. According to a Pfizer spokesperson, no patients have received commercialized Beqvez treatment since its FDA approval in April 2024.

Pfizer will notify patients and providers who are in the process of qualifying for treatment, and has committed to continue supporting patients receiving the drug in clinical trials. Pfizer’s website shows that the company currently has no other gene therapy projects in commercial or clinical stages, and a spokesperson confirmed that Pfizer currently has no active gene therapy projects.

Pfizer Gradually Exits the Gene Therapy Field

Pfizer's gradual exit from the gene therapy field can be traced back a few months. Two months ago, Pfizer terminated the hemophilia A gene therapy project giroctocogene fitelparvovec, which was being co-developed with Sangamo Therapeutics, despite the project showing positive results in phase three trials.

In 2023, Pfizer sold its preclinical gene therapy programs and related technologies to Alexion, AstraZeneca's rare disease unit, for up to $1 billion as part of a broader transformation of the company. Additionally, last year, Pfizer's Duchenne muscular dystrophy gene therapy candidate unexpectedly failed in Phase III trials, leading the company to abandon the project and lay off more than 200 employees at its production facility in North Carolina.

Challenges in the Field of Gene Therapy

Pfizer's experience reflects the challenges faced by other hemophilia gene therapy developers. The market promotion of CSL's Hemophilia B gene therapy, Hemgenix, has also been less than expected. CSL CEO Paul McKenzie pointed out in a conference call that the fragmented healthcare system in the United States is the main reason for the slow rollout.

Similarly, BioMarin's hemophilia A gene therapy Roctavian also faces slow sales. Despite Roctavian receiving FDA approval in June 2023 and launching at a price of $2.9 million, its sales in 2024 were only $26 million.

Future Plans

Pfizer acquired Beqvez from Spark Therapeutics in 2014, which was later acquired by Roche. With the termination of the collaboration with Sangamo and the discontinuation of Beqvez, Pfizer has shifted its hemophilia resources to Hympavzi, a weekly non-factor antibody drug that received FDA approval last year for treating Hemophilia A and B.

In addition, Pfizer has also partnered with Beam Therapeutics to focus on mRNA-delivered gene editing technology rather than non-viral vector gene therapy. Despite Pfizer’s $300 million investment in this collaboration, no projects have been officially adopted so far.

Beqvez is the second blood disease product that Pfizer has withdrawn from the global market in less than half a year. In September last year, Pfizer decided to stop distributing the sickle cell disease treatment drug Oxbryta due to concerning results from the Phase III trial.

Analysis and Reflection

Pfizer's Exit from Gene Therapy Sparks Broad Discussion on Commercialization Challenges

Moreover, the success of gene therapy depends not only on the technology itself but also on various factors such as the healthcare system, payment models, and patient acceptance. Pfizer's withdrawal reminds us that the commercialization path of gene therapy is still full of challenges and requires joint efforts from both inside and outside the industry to overcome.

In the future, the field of gene therapy may see more cooperation and integration to address these challenges. At the same time, policymakers and healthcare systems also need to explore new payment models and regulatory frameworks to support the sustainable development of gene therapy.