Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez.
The New York pharma is ending global development and commercialization of Beqvez less than a year after an FDA approval for the gene therapy to treat hemophilia B. The one-time treatment carried a list price of $3.5 million per person.
Several reasons led to the discontinuation, including limited interest from patients and doctors toward hemophilia gene therapies to date, a Pfizer spokesperson told Fierce Pharma in a statement.
No patients seem to have received commercial Beqvez since its FDA nod in April 2024. The Pfizer spokesperson said the company will communicate the news to patients and providers that are in the treatment qualification process, adding that the company remains committed to supporting those who received the med in any clinical trial.
Following Beqvez’s exit from the market, Pfizer has no commercial or clinical-stage gene therapies left in the works, according to its website. The spokesperson confirmed that the company doesn’t have any active gene therapy programs at the moment.
Step by step, Pfizer has been pulling out of the gene therapy field—either purposefully or inadvertently.
The move to pull Beqvez off the market comes two months after Pfizer terminated a hemophilia A gene therapy pact with Sangamo Therapeutics even though the project involved, giroctocogene fitelparvovec, passed muster in a phase 3 trial.
Prior to that, in a deal worth up to $1 billion signed in 2023, Pfizer sold its preclinical gene therapy programs and related technologies to AstraZeneca’s rare disease unit Alexion as part of a broader pivot away from early-stage rare disease R&D.
Then, last year, the company’s Duchenne muscular dystrophy gene therapy candidate unexpectedly flopped in a phase 3 test. Pfizer abandoned the program, took a $230 million impairment charge and laid off more than 200 staffers at a manufacturing facility in Sanford, North Carolina, which was producing the DMD prospect.
The Sanford site is also responsible for making Beqvez. Pfizer is evaluating potential job cuts related to the product termination, the spokesperson said, noting that “any related decisions will be handled individually and in a timely manner.”
Overall, the company doesn’t expect the latest move to have a material impact on the operations at the facility, the spokesperson said.
Pfizer’s experience mirrors the struggles of other hemophilia gene therapy players. CSL’s first-to-market rival hemophilia B gene therapy, Hemgenix, has also faced a slower-than-expected launch. During a conference call in August, CSL CEO Paul McKenzie attributed the drug's low market penetration to a fragmented U.S. healthcare system.
The challenge is that most hemophilia treatment centers are independent from hospital systems but hospitals require advanced therapies like gene therapies to be managed through their in-house clinical pharmacies, McKenzie explained on another call in February. As a result, when Hemgenix launched, hemophilia centers and hospitals needed to rework their relationships and figure out how to divvy up the appropriate accountabilities and financial benefits, he said.
On that February call, McKenzie described Hemgenix’s uptake as “accelerating” as CSL “slowly but steadily find[s] a way through the complexities of the U.S. healthcare system.” Nevertheless, the Australian company has recently decided to shutter its cell and gene therapy R&D hub in Pasadena, California, as it deprioritizes ex vivo lentiviral-based gene therapy work in general.
Similarly, BioMarin has also faced difficulties with its hemophilia A gene therapy Roctavian. While working through a slow sales ramp, the California biotech last year decided to narrow its commercial focus of Roctavian to three markets, laying off employees as a result.
Approved by the FDA in June 2023 and a launched at a list price of $2.9 million, Roctavian only brought in $26 million in sales in 2024. In contrast, Roche’s hemophilia A antibody drug Hemlibra booked 4.5 billion Swiss francs ($5 billion) in global revenue, good for 12% growth year over year.
Pfizer got Beqvez in 2014 from Spark Therapeutics. The gene therapy biotech was later acquired by Roche, which has had its own struggles with the modality in hemophilia.
With its Sangamo partnership terminated and Beqvez being discontinued, Pfizer is shifting its hemophilia resources to Hympavzi, a once-weekly nonfactor antibody drug that the FDA approved last year for hemophilia A and B.
As for Pfizer’s broader gene therapy plan, the company has been involved in a research collaboration with Beam Therapeutics focused on mRNA-delivered gene editing rather than viral vector-based gene therapy. The deal saw Pfizer put down $300 million upfront for Beam’s base editing technology in search for up to three candidates for rare genetic diseases of the liver, muscle and central nervous system.
The four-year discovery pact is coming to its final year this year, but there’s been no word of any project that Pfizer has decided to pick up. The prospects don't look too promising for Beam given Pfizer’s recent retreat from early-stage rare disease.
Beqvez marks the second blood disorder product that Pfizer is pulling from global markets in less than half a year. Back in September, the company decided to stop distributing the sickle cell disease treatment Oxbryta after a phase 3 trial turned up some concerning results.