Philly biotech gets FDA ok to review gene therapy treatment

Philadelphia drug developer Spark Therapeutics said Monday the Food and Drug Administration has accepted its biologics license application and granted “priority review” for its lead drug candidate to treat rare inherited  blindness.

If approved, the treatment would be the first gene therapy for a genetic disease in the United States.

Spark’s treatment, called voretigene neparvovec, streams genes directly into the retina of the eye. It has been granted priority review by the FDA because it treats a medical condition where no “adequate therapy” exists, the company said.

The time frame for possible approval is about six months, around Jan. 12, 2018.

Spark was spun out of Children’s Hospital of Philadelphia, based on decades of research led by Katherine A. High, now Spark’s President and Chief Scientific Officer.

“It’s really an exciting moment for medicine,” said Spark chief executive officer Jeffrey Marrazzo, noting that an FDA panel last week reviewed an experimental cellular therapy being developed by Novartis and the University of Pennsylvania to treat cancer. The original study for the CAR-T cell technology was done at Children’s Hospital of  Philadelphia, he said.

Spark does not have confirmation, but expects the FDA may convene an advisory meeting of medical experts in the fall to consider the company’s data from three clinical trials, which enrolled 41 participants.

In a late-stage, Phase 3 study of 20 people, 93 percent had vision improvement and “saw restoration of aspects of their functional vision,” Marrazzo said.  Patients in an earlier Phase 1 trial have been followed now for four years and continue to maintain their original vision improvement, he said.

Spark’s treatment injects…

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