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FDA approves $3.5m gene therapy for adults with haemophilia B

BMJ 2022; 379 doi: https://doi.org/10.1136/bmj.o2858 (Published 25 November 2022) Cite this as: BMJ 2022;379:o2858
  1. Janice Hopkins Tanne
  1. New York

The US Food and Drug Administration has approved the first gene therapy to treat adults with haemophilia B under the agency’s priority review and breakthrough therapy processes.1

Marketed as Hemgenix, etranacogene dezaparvovec-drib is based on an adeno-associated virus vector. The FDA said it is given as a single dose by intravenous infusion. The gene is expressed in the liver to produce factor IX protein, to increase blood concentrations of factor IX and thus limit bleeding episodes.

The FDA said it is indicated for the treatment of adults with a single gene disorder: haemophilia B …

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