An experimental Spark Therapeutics gene therapy intended to treat rare eye diseases that lead to blindness has won the unanimous backing of an advisory panel to the FDA.
Following a daylong hearing that included presentations from Philadelphia-based Spark (NASDAQ: [[ticker:ONCE]]), FDA staff, ophthalmologists, and patients, the panel of independent eye experts voted 16-0 on the question of whether the treatment’s benefits outweigh its risks. Spark CEO Jeff Marrazzo (pictured above, right) called the vote an “historic moment.”
“This is the first ever gene therapy of its kind to get to this stage of development, to get to an [FDA advisory committee],” he said. “To get a unanimous recommendation of approval is pretty significant.”
If the FDA approves the Spark drug, voretigene neparvovec (Luxturna), it would mark the first therapy approved by the agency that replaces a faulty gene with a working one, offering a long-lasting treatment, and perhaps even a cure. Though the panel’s vote is advisory, FDA staff do consider the discussion and recommendation of these committees. An FDA decision is expected by Jan. 12.
Trading of Spark shares was halted during Thursday’s hearing. But in after hours trading, Spark shares rose 7.2 percent to $92.43 per share.
The drug is meant to whether the therapy that Spark intends to be a one-time treatment might end up needing additional doses. Marrazzo said that Spark has data extending four years showing that patients continue to benefit from their treatment.
“The data suggest that it could be very long-lasting, if not lifelong,” he said.
Photo by Spark Therapeutics.
