Breakthrough status for Pfizer/Spark haemophilia gene therapy

Post Date: 
22 July 2016

fizer and Spark Therapeutics say their investigational haemophilia B drug SPK-9001 has been awarded breakthrough therapy status by the US Food and Drug Administration.

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

Breakthrough therapy designation is intended to speed up the development and regulatory review of drugs to treat a serious or life-threatening disease or condition, and is awarded on the back of preliminary clinical evidence suggesting a potentially substantial improvement over existing therapies on at least one clinically significant endpoint.

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