Pfizer, Spark Therapeutics Get FDA Breakthrough Status for Hemophilia B DrugBy
Pfizer and Spark Therapeutics, a Philadelphia, Pennsylvania-based gene therapy company, have been granted breakthrough therapy designation by the US Food and Drug Administration for SPK-9001, the companies' lead investigational candidate for hemophilia B. The compound is a bio-engineered adeno-associated virus (AAV) capsid that is in an ongoing Phase I/II trial. Breakthrough therapy designation is intended to expedite development and FDA review.
Spark Therapeutics and Pfizer entered into a collaboration in 2014 for the SPK-FIX program, including SPK-9001, under which Spark Therapeutics is responsible for conducting all Phase I/II studies for any product candidates while Pfizer will assume responsibility for pivotal studies, any regulatory activities and potential global commercialization of any products that may result from the collaboration. Spark Therapeutics’ proprietary technology platform for selecting, designing, manufacturing and formulating highly optimized gene therapies was applied to developing compounds in the SPK-FIX program.