FDA nod for novel gene therapy to treat a rare form of inherited vision loss

In what is seen as a historic move, the US Food and Drug Administration has approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may result in blindness.

US biotech firm Spark Therapeutics’ (Nasdaq: ONCE) Luxturna is the first directly administered gene therapy approved in the US that targets a disease caused by mutations in a specific gene, and the decision comes well ahead of the anticipated date of mid-January 2018.

The approval was widely expected, given a unanimous recommendation vote by an FDA advisory panel in October. The company’s shares jumped as much as 6.9% to $52.39 in early trading on Tuesday following the announcement, but later fell back.