Pfizer’s First Gene Therapy Gets US Nod for Clotting Ailment

  • Beqvez will cost $3.5 million, mirroring CSL Behring’s product
  • Therapy reduced bleeding 71% compared with standard treatment
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Pfizer Inc. won US approval for its first gene therapy, expanding into a promising field of medicine that has proved to be a challenging business.

The treatment, called Beqvez, gained clearance for patients with hemophilia B, a rare bleeding disorder, Pfizer said Friday in a statement. Pfizer’s medicine is a one-time therapy designed to insert a correct copy of the mutated gene that causes hemophilia B, preventing the dangerous bleeding episodes that mark the disease.