Pfizer/Spark team gets a ‘breakthrough’ in gene therapy alliance

Print 26 July 2016
John Carroll / ENDPOINTS

A couple of years ago, Pfizer set up a new group to tackle the hot gene therapy field, drafting Michael Linden from King’s College to get it going. Right off the bat, the pharma giant also teamed with Spark Therapeutics – a hot player in the gene therapy field – to partner on an experimental program for hemophilia B. And now the partners can boast of a ‘breakthrough’ drug designation at the FDA to help speed their work along.

SPK-9001 is another AAV vehicle carrying a human factor IX variant now in a Phase I/II study.

This is Spark’s second BTD. It won another for voretigene neparvovec, in development for inherited retinal disease (IRD) caused by mutations in the RPE65 gene. That therapy appeared to be headed to become the first new gene therapy to be approved in the U.S. after acing a Phase III study last fall.

Pfizer paid Spark $20 million to team up in 2014, with another $260 million set aside for their progress.

“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,” said Spark CEO Jeffrey D. Marrazzo in a statement.

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