Spark Therapeutics Announces Positive CHMP Opinion for One-time Gene Therapy LUXTURNA (voretigene neparvovec) in the European Union

LUXTURNA would be first gene therapy for a genetic disease approved in both U.S. and EU

LUXTURNA would be first and only gene therapy approved in EU to treat patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells, a condition leading to total blindness in most patients

Positive opinion based on robust data package supporting safety, efficacy and durability of response for up to three years after one-time treatment with LUXTURNA

PHILADELPHIA, Sept. 21, 2018 (GLOBE NEWSWIRE) -- Spark Therapeutics (NASDAQ: ONCE), a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending approval of LUXTURNA (voretigene neparvovec), a one-time gene therapy for the treatment of adult and pediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.

"We are encouraged by today's decision, which further affirms our pioneering clinical program and brings LUXTURNA one step closer to patients with inherited retinal disease (IRD) caused by mutations in both copies of the RPE65 gene in the European Union," said Katherine A. High, M.D., president and head of research & development at Spark Therapeutics. "Along with Novartis, we look forward to continuing our productive, ongoing dialogue with EMA toward potential marketing authorization to make LUXTURNA the first and only treatment for appropriate patients with this inherited retinal disease (IRD) in the European Union."

The positive CHMP opinion is based on data from a Phase 1 clinical trial, its follow-up trial and a Phase 3 trial that together enrolled 43 participants with inherited retinal disease caused by mutations on both copies of the RPE65 gene. The Phase 3 trial was the first randomized, controlled Phase 3 gene therapy trial for a genetic disease. Spark Therapeutics has received orphan product designation for LUXTURNA from EMA for the treatment of inherited retinal dystrophies.

"Inherited retinal diseases are a heterogenous group of degenerative conditions that represent the major cause of blindness in childhood and active working life. This opinion represents a hopeful milestone for current and future patients who may ultimately benefit from gene therapy," said Christina Fasser, president of Retina International, an umbrella organization of more than 43 patient organizations worldwide promoting research to find treatments for inherited retinal degenerative diseases.