FDA/CDC

Gene therapy for hemophilia A gets fast-track review


 

The investigational gene therapy SPK-8011 for the treatment of hemophilia A is getting an accelerated review at the Food and Drug Administration, according to Spark Therapeutics.

The FDA granted breakthrough therapy designation to the gene therapy product in February and orphan drug status in January.

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The company presented early phase 1/2 clinical trial data at the annual meeting of the American Society of Hematology. The SPK-8011 data included the first four participants who had been followed for at least 12 weeks post infusion. As of the Dec. 6, 2017, cutoff date, there was a 100% reduction in the annual bleeding rate and 98% reduction in annualized infusion rate after week 4, according to Spark Therapeutics.

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