Pfizer and Spark Therapeutics granted FDA breakthrough designation for haemophilia drug
pharmafile | July 22, 2016 | News story | Medical Communications, Research and Development | FDA, Pfizer, Spark Therapeutics, haemophilia B, spk-9001
Pfizer and Spark Therapeutics have announced that the US Food and Drug Administration (FDA) has granted a breakthrough therapy designation to SPK-9001, which is currently in development for the treatment of haemophilia B.
The pharma giant announced its partnership with Spark in late 2014, and has since worked together on the SPK-FIX program which attempts to leverage a long history of haemophilia gene therapy research and clinical development conducted by Spark.
The pair presented early data on SPK-9001, a bio-engineered adeno-associated virus, in May which showed a consistent increase in factor XI levels through the first four weeks post-administration of the drug.
The strength of this data will surely have helped in gaining this breakthrough therapy designation from the FDA, which requires preliminary clinical evidence that the investigational therapy may offer substantial improvement over existing therapies on at least one clinically significant endpoint.
Jeffrey Marrazzo, CEO at Spark Therapeutics, says: “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 haemophilia B through a potentially one-time, intravenous administration of a highly optimised gene therapy.”
Sean Murray
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