FDA accepts biologics license application for voretigene neparvovec
The FDA has accepted a biologics license application and granted priority review for voretigene neparvovec, according to a press release from Spark Therapeutics.
An investigational gene therapy candidate for vision loss due to biallelic RPE65-mediated inherited retinal disease (IRD), voretigene neparvovec, which has the proposed trade name of Luxturna, could be both the first gene therapy for a genetic disease in the United States and the first pharmacologic treatment for IRD, the release said.
A PDUFA date for the treatment, which is intended to be administered one time per eye, has been set for Jan. 12, 2018.
“FDA acceptance for filing of our BLA for Luxturna is an important development for people living with RPE65-mediated IRD, a significant milestone for the gene therapy field and a strong testament to the dedication of our collaborators and employees,” Jeffrey D. Marrazzo, CEO of Spark Therapeutics, said in the release. “As we work closely with the FDA in the months ahead, we will remain steadfast in our commitment to bring this important investigational therapy to people living with RPE65-mediated IRD who currently have no pharmacologic treatment options.”