FDA grants SPK-9001 breakthrough therapy designation

Spark Therapeutics and Pfizer Inc. recently announced that SPK-9001 has been granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA). 

SPK-9001 is the lead investigational candidate in the two companies’ joint SPK-FIX program. SPK-9001, a novel bioengineered adeno-associated virus (AAV), is currently being developed to treat hemophilia B and is currently undergoing a Phase 1/2 trial.

“We are extremely pleased to have been granted breakthrough therapy designation for SPK-9001, which has shown early promise in achieving our goal of eliminating the need for regular infusions to control and prevent bleeding episodes in patients with hemophilia B through a potentially one-time, intravenous administration of a highly optimized gene therapy,” CEO of Spark Therapeutics Jeffrey D. Marrazzo said. “Together with Pfizer, we look forward to working closely with the FDA to bring SPK-9001 to patients as quickly and responsibly as possible.”

The designation of breakthrough therapy is a way of speeding up the development of experimental drugs as well as the FDA’s review of drugs that are designed to treat conditions and diseases that are serious or life-threatening. In order to receive a breakthrough therapy designation, a treatment must show that it is an improvement to current therapies.