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

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.1Marketed 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 (congenital deficiency of factor IX, which is needed to produce blood clots). These patients currently use factor IX prophylaxis therapy or have current or historical life threatening haemorrhage or have repeated, serious, spontaneous bleeding episodes.The one time treatment will cost $3.5m (£2.9m; €3.4m). CSL Behring, the drug’s manufacturer, said the treatment would reduce healthcare costs because patients would…
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