Spark Therapeutics Submits Marketing Authorization Application to European Medicines Agency for Investigational LUXTURNA(TM) (voretigene neparvovec)

Potential for first gene therapy for a genetic disease to be approved in both the U.S. and EU

PHILADELPHIA, July 31, 2017 (GLOBE NEWSWIRE) -- Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, announced today that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for LUXTURNA(TM), the proposed trade name for voretigene neparvovec, an investigational, one-time gene therapy for the treatment of patients with vision loss due to Leber congenital amaurosis or retinitis pigmentosa caused by confirmed biallelic RPE65 mutations. The MAA includes data from three clinical trials that enrolled 41 participants with RPE65-mediated inherited retinal dystrophy (IRD), including the first randomized, controlled Phase 3 trial for a gene therapy for a genetic disease.

Once EMA has validated the application, the review period will begin. Spark Therapeutics has previously received orphan product designations for LUXTURNA from EMA for the treatment of both Leber congenital amaurosis and retinitis pigmentosa.

"Today's announcement represents an important moment in the effort to treat blindness caused by inherited retinal degenerative diseases," said Christina Fasser, president of Retina International, an umbrella organization of more than 43 patient organizations world-wide promoting research in view to find a cure to inherited retinal degenerative diseases. "We are excited about the potential of this application to bring the first gene therapy to patients with this form of IRD."

The investigational therapy is under Priority Review with the U.S. Food and Drug Administration, with an assigned Prescription Drug User Fee Act (PDUFA) date of Jan. 12, 2018.

"With LUXTURNA now in regulatory review on both sides of the Atlantic, we are building out our medical and commercial infrastructure to prepare to bring investigational LUXTURNA to patients in the U.S. and Europe," said John Furey, chief operating officer of Spark Therapeutics. "For the first time, these individuals, who eventually will progress to complete blindness, have hope for a potential treatment option that may restore their vision. We remain steadfast in our commitment to bring this important investigational therapy to patients in the EU with vision loss due to Leber congenital amaurosis or retinitis pigmentosa caused by confirmed biallelic RPE65 mutations."