Pfizer offers warranty for new haemophilia B therapy Beqvez

Pfizer has said it will launch its just-approved haemophilia B gene therapy Beqvez with a warranty programme that will provide “greater certainty” to payers, after setting a price directly in line with a rival treatment.

Beqvez (fidanacogene elaparvovec) was approved by the FDA on Friday as a one-time therapy for adults with moderate to severe haemophilia B who need factor IX replacement therapy, but still suffer bleeding episodes. It will be priced at $3.5 million – the same as CSL Behring and uniQure’s Hemgenix, which was cleared in the US towards the end of 2022.

The warranty will be based on the durability of patients’ response to the gene therapy, providing insurance against the risk that the treatment does not work as expected, said Pfizer, which plans to launch Beqvez later this quarter.

It is the pharma giant’s first gene therapy, stemming from an alliance with Spark Therapeutics (now part of Roche) a decade ago, and has also been approved in Canada. It is currently under review by regulators in Europe.

Pfizer said in a statement that the one-off treatment for the bleeding disorder provides value for money because it avoids the need for regular prophylactic infusions of factor IX replacement and provides sustained bleed protection, “potentially avoiding years of treatment burden.”

The cost of infusions is typically around $300,000 a year in the US, according to figures from the National Bleeding Disorders Foundation (NBDF), and can be twice that for severe disease. If a patient has inhibitors – an immune response to replacement clotting factors – the annual cost can rise above $1 million.

For patients, the importance of potentially becoming free of regular infusions and bleeding complications cannot be overstated.

“Many people with haemophilia B struggle with the commitment and lifestyle disruption of regular infusions, as well as spontaneous bleeding episodes, which can lead to painful joint damage and mobility issues,” said Adam Cuker, director of Penn Medicine’s Comprehensive and Haemophilia Thrombosis Programme.

Beqvez “has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term,” he added.

Pfizer has previously reported data from a small group of 15 patients who received Beqvez in a phase 1/2 trial, showing they had no need for prophylactic factor IX therapy for five years after dosing. Four of them had undergone surgeries during that period, with no bleeding complications or any need for replacement therapy.

The company is also following patients from the phase 3 BENEGENE-2 study that underpinned Beqvez’s approval for up to six years.

An estimated 38,000 patients are living with haemophilia B worldwide, according to the World Federation of Haemophilia. As the gene is carried on the X chromosome, haemophilia is almost entirely a male disease.

Pfizer, meanwhile, is also developing gene therapies for haemophilia A and Duchenne muscular dystrophy (DMD), and its pipeline includes marstacimab, a potential subcutaneous therapy option for both haemophilia A and B, with or without inhibitors.

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