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Spark Therapeutics (ONCE), Pfizer (PFE) Hemophilia Therapy gets FDA Breakthrough Status

July 21, 2016 9:01am

SPK-9001, is being developed for the treatment of hemophilia B, ONCE is UP +$0.57 or +1.05% to $55.00

Spark Therapeutics (ONCE) and Pfizer Inc. (PFE) 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 P1/2 trial as a potential one-time therapy.

The Bottom Line: 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.

ONCE had seen sustained factor IX Levels in 4 Patients

ONCE closed at $54.43 and is UP in the pre-market +$0.57

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