JAN 25, 2018
Comprehensive Ophthalmology, Pediatric Ophth/Strabismus, Retina/Vitreous
Swiss-based Novartis Pharmaceuticals has acquired rights to commercialize Luxturna—Spark Therapeutics’ gene therapy for inherited retinal dystrophy—outside of the United States. Spark will retain exclusive rights to Luxturna in the U.S., however.
Luxturna is the first gene therapy approved in the U.S. to restore light perception in patients with biallelic RPE65 mutations. Spark agreed to pursue European approval, and filed for a marketing authorization with the European Medicines Agency (EMA) on July 31, 2017. In addition to paying $105 million up front, Novartis will make milestone payments up to $65 million if EMA approval is granted.
Spark hopes the move will help ensure that more patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy who live outside the U.S. have access to voretigene neparvovec, according to Dan Faga, chief business officer of Spark Therapeutics.
“We intend to use the proceeds from this transaction to continue to develop our robust pipeline of investigational gene therapies to create a path to a world where no life is limited by genetic disease,” Faga said.
The announcement caps a busy period for the Philadelphia-based biotech company, and comes about 1 month after FDA’s landmark approval of Luxturna. Pricing for the gene therapy—$425,000 per eye—was announced in early January.
Novartis, on the other hand, is days ahead of a significant change in leadership. Vasant Narasimhan, MD, currently global head of drug development and chief medical officer, will assume the role of CEO effective February 1, 2018. He replaces Joseph Jimenez, who will step down after leading Novartis for 8 years.