Spark gets FDA's first in vivo gene therapy approval

In its first approval of an in vivo gene therapy, FDA approved Luxturna voretigene neparvovec from Spark Therapeutics Inc. (NASDAQ:ONCE) to treat biallelic retinal pigment epithelium-specific protein 65kDa (RPE65) mutation-associated retinal dystrophy. The approval comes ahead of its Jan. 12 PDUFA date.

Spokesperson Monique da Silva told BioCentury that Spark will announce Luxturna's pricing in early January. On its 3Q17 financial results call last month, Spark said its economic modeling for Luxturna valued the gene therapy at over $1 million (see BioCentury, Nov. 10)...