FDA Grants Breakthrough Therapy Designation For Spark, Pfizer's SPK-9001

(RTTNews) - Spark Therapeutics (ONCE) and Pfizer Inc. (PFE) said that the U.S. Food and Drug Administration or FDA has granted breakthrough therapy designation to SPK-9001, the lead investigational candidate in the two companies' SPK-FIXprogram, in development for the treatment of hemophilia B.

SPK-9001, a novel bio-engineered adeno-associated virus or 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 meant 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 has previously received breakthrough therapy designation for voretigene neparvovec, which is being developed for the potential treatment of inherited retinal disease or IRD caused by mutations in the RPE65 gene.