Pfizer drops Beqvez, leaving its gene therapy cupboard bare
In another sign of the challenges facing gene therapy developers, Pfizer has abandoned its haemophilia B treatment Beqvez in all world markets, saying weak demand made the business non-viable.
The decision comes after Pfizer ended a partnership with Sangamo Therapeutics on a haemophilia A gene therapy candidate, despite positive phase 3 results, leaving it with no active gene therapy programmes in its development pipeline.
It also follows the decision by gene therapy pioneer bluebird bio to sell itself to private equity companies for a bargain price after struggling to build sales momentum for its three FDA-approved gene therapies.
Pfizer said patients and doctors had shown "limited interest" in Beqvez (fidanacogene elaparvovec) since it was approved for marketing in the US, EU, and Canada last year, and there were signs of that disillusionment last week when the company withdrew a marketing application to sell the gene therapy in Japan.
The gene therapy launched in the US last year with a $3.5 million price tag as a one-time therapy for adults with moderate to severe haemophilia B who need factor IX (FIX) replacement therapy, but who still have bleeding episodes.
Pfizer implemented a warranty programme that provided money back to insurers if the treatment did not work as expected, but it seems that was not enough to encourage uptake – despite the high cost of FIX infusions that can run to around $300,000 a year.
Other haemophilia gene therapy developers – BioMarin and CSL – have also reported slow uptake with their products, and BioMarin said recently it will not try to expand access to its Roctavian (valoctocogene roxaparvovec) haemophilia A product beyond the first three markets where it already has reimbursement approval – the US, Germany, and Italy.
CSL, meanwhile, has said that its Hemgenix haemophilia B therapy is rolling out a lot slower than expected due in part to complexities in the US healthcare system.
Beqvez was Pfizer's first gene therapy, stemming from an alliance with Spark Therapeutics (now part of Roche) more than a decade ago, at a time when it was actively building a presence in gene therapy, with an ambition to become "the industry leader" in the category.
It also bought an option on a liver disease therapy developed by Vivet Therapeutics and bought Bamboo Therapeutics to provide a viral vector platform for gene therapy delivery and early-stage candidates for diseases, including one for Duchenne muscular dystrophy that failed a phase 3 study last year.
In 2023, however, it sold off its early-stage rare disease gene therapy portfolio to AstraZeneca's Alexion unit in a $1 billion deal, in an early sign that it was exiting the category.
