Pfizer has received approval from the US Food and Drug Administration (FDA) for its one-time gene therapy BEQVEZ (fidanacogene elaparvovec-dzkt) to treat adults with moderate to severe haemophilia B.

Haemophilia B is a rare genetic disorder characterised by insufficient blood clotting due to a deficiency in factor IX (FIX), leading to prolonged and more frequent bleeding.

BEQVEZ enables patients to produce FIX internally, potentially eliminating the need for regular prophylactic infusions.

Its approval is based on results from the pivotal Phase III BENEGENE-2 trial.

The study demonstrated that BEQVEZ significantly reduced the annualised bleeding rate in participants compared to their previous FIX prophylaxis regimen.

It enrolled 45 subjects with moderately severe to severe haemophilia B, who received a single intravenous infusion of BEQVEZ.

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Participants in the BENEGENE-2 study will be monitored for up to 15 years to assess the long-term safety and efficacy of the therapy. This includes a six-year follow-up in the Phase III study and an additional nine years in a separate Phase III study.

Previous Phase I/IIa studies and their long-term follow-ups have already provided data for up to six years.

As BEQVEZ awaits review by the European Medicines Agency, it has already received regulatory approval in Canada.

Pfizer chief US commercial officer and executive vice-president Aamir Malik stated: “This milestone is a testament to Pfizer’s continued effort to advance the standard of care for people living with haemophilia, with the delivery of a medicine that has the potential to offer both long-term bleed protection and value to the healthcare system because of its one-time administration.

“We are leveraging our expertise that comes with more than 40 years of experience in the haemophilia space, and are proactively working with treatment centres, payers and the haemophilia community to appropriately help ensure the healthcare system is prepared to readily deliver BEQVEZ to the patients who can benefit from it.”

The European Commission recently granted marketing authorisation for Pfizer’s Emblaveo (aztreonam-avibactam) for treating adults with multidrug-resistant infections where treatment options are limited.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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