FDA approves Pfizer’s first gene therapy for rare inherited bleeding disorder

CNBC April 26, 2024
Annika Kim Constantino

Key Points

– The Food and Drug Administration approved Pfizer’s treatment for a rare genetic bleeding disorder, making it the company’s first gene therapy to win clearance in the U.S.

– The agency greenlighted the drug, which will be marketed as Beqvez, for adults with moderate to severe hemophilia B.

– Pfizer said the treatment will be available by prescription to eligible patients this quarter and has a hefty $3.5 million price tag before insurance and other rebates.

The Food and Drug Administration on Friday announced that the agency approved Pfizer‘s treatment for a rare genetic bleeding disorder, making it the company’s first gene therapy to win clearance in the U.S.

The agency greenlighted the drug, which...

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