21 July 2016 - Spark Therapeutics and Pfizer announced today that the U.S. FDA has granted breakthrough therapy designation to SPK-9001, the lead investigational candidate in the companies’ SPK-FIX program, in development for the treatment of hemophilia B.

SPK-9001, a novel bio-engineered adeno-associated virus (AAV) capsid expressing a codon-optimized, high-activity human factor IX variant, is being investigated in an ongoing Phase 1/2 trial as a potential one-time therapy.

Breakthrough therapy designation is intended to expedite the development and FDA review of drugs to treat a serious or life-threatening disease or condition. The designation requires preliminary clinical evidence that the investigational therapy may offer substantial improvement over existing therapies on at least one clinically significant endpoint.

In addition to SPK-9001, Spark Therapeutics previously received breakthrough therapy designation for voretigene neparvovec, which is being developed for the potential treatment of inherited retinal disease (IRD) caused by mutations in the RPE65 gene.

Read Pfizer press release