Pfizer is ending the development and commercialization of its hemophilia B gene therapy Beqvez, the company confirmed in an emailed statement to Endpoints News.
The company cited “limited interest patients and their doctors have demonstrated in hemophilia gene therapies to date.” The spokesperson confirmed that zero patients had received the commercial drug since approval.
“We have informed our partners, Roche/Spark, and are discussing the best next steps for the program,” the spokesperson added.
It marks an abrupt end for the gene therapy for the rare, inherited bleeding disorder. Pfizer gained FDA approval for the one-time treatment last April, and the New York pharma giant rolled it out at a price of $3.5 million.
The drugmaker didn’t disclose any updates on the gene therapy during its recent earnings. A decade ago, it had licensed Beqvez from Spark Therapeutics, which is now owned by Roche.
Kim Phelan, CEO of the Coalition for Hemophilia B, said the nonprofit was “disappointed to hear of this discontinuation,” but that they “respect Pfizer’s decision as they evaluate their portfolio and future direction.”
“Importantly, we remain hopeful and reassured that individuals with hemophilia B still have access to an approved gene therapy option,” Phelan said in a statement, referring to CSL Behring’s Hemgenix. CSL has struggled to gain traction for the multimillion-dollar treatment.
The pullback casts a new pall over the blood disorder landscape. Two months ago, Pfizer axed its partnership on another gene therapy, a hemophilia A treatment from Sangamo Therapeutics that cleared a Phase 3 last summer. The drugmaker also pulled another blood disorder drug, the sickle cell treatment Oxbryta, last fall after saying the “totality of clinical data” showed the overall benefit of the drug “no longer outweighs the risk.”
“We will communicate to those Beqvez patients who are in the qualification process and their providers who have initiated the qualification process and follow their desired pathway through our decision,” the Pfizer spokesperson said in an email. “For any patient who has been treated with Beqvez in any clinical trial, Pfizer remains committed to supporting the clinical trial sites and patients with regard to clinical trial follow-up and patient and trial support.”
Pfizer markets another drug for hemophilia A and B. In October, the FDA greenlit the company’s once-weekly injectable, branded as Hympavzi.
Check out our AAV CDMO service to expedite your gene therapy research
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
Related News
Nanoscope Therapeutics Publishes Breakthrough Clinical Data Demonstrating Significant Vision Restoration in Retinitis Pigmentosa with Novel AAV-based MCO-010 Gene Therapy
Nanoscope Therapeutics has announced the publication of highly encouraging Phase 1/2a clinical trial data in the esteemed journal Molecular Therapy, showcasing significant vision restoration in patients suffering from retinitis pigmentosa (RP), a debilitating...
Capsida’s AAV CAP-004 Gene Therapy Shows Promise for Friedreich’s Ataxia in Primate Study at MDA Conference
Capsida Biotherapeutics' experimental gene therapy, CAP-004, for Friedreich's ataxia (FA), has demonstrated promising preclinical results in nonhuman primates. Research presented by Capsida at the 2025 MDA Clinical & Scientific Conference showed that a single...
FDA Grants Fast Track to Sanofi’s Chlamydia mRNA Vaccine
Sanofi is making significant strides in the fight against chlamydia, the most common sexually transmitted bacterial infection, for which there are currently no approved preventative vaccines. The French pharmaceutical group has recently been granted a fast-track...
Epicrispr Biotechnologies Announces $68M Series B to Advance First-in-Class FSHD Epigenetic Therapy to Clinic
Epicrispr Biotechnologies today announced the first close of its Series B financing, securing $68 million to support the clinical development of EPI-321, a first-in-class, disease-modifying therapy for facioscapulohumeral muscular dystrophy (FSHD), a genetic...
Related Services
AAV Packaging Services
READ MORE
Off-the-Shelf AAV Products
READ MORE
