Novartis Luxturna Gene Therapy for restoring Eyesight in Rare retinal diseases gets European Approval

ZURICH: Novartis on Friday said it had received European Union approval for its Luxturna gene therapy to restore sight to people with a rare retinal disease. Spark Therapeutics will manufacture and supply Luxturna for the company, while Novartis has exclusive rights to pursue development, registration and commercialization in all other countries outside the U.S.

Luxturna is a one-time treatment for children and adults who have suffered impaired vision from an early age due to mutations in both copies of the RPE65 gene. Such mutations, which can lead to blindness, affect roughly 1 in 200,000 people, according to Novartis.

Novartis International AG is a Swiss multinational pharmaceutical company based in Basel, Switzerland. It is one of the largest pharmaceutical companies by both market capitalization and sales.

Novartis is a full member of the European Federation of Pharmaceutical Industries and Associations (EFPIA),the International Federation of Pharmaceutical Manufacturers and Associations (IFPMA), and the Pharmaceutical Research and Manufacturers of America (PhRMA)

The authorization is valid in all 28 member states of the EU, as well as Iceland, Liechtenstein and Norway, Novartis said. Luxturna was developed and is commercialized in the United States by Spark Therapeutics.

In January 2018, Spark Therapeutics entered into a licensing agreement with Novartis to commercialize Luxturna when approved in Europe and all other markets outside the U.S. Under the licensing agreement, Novartis has exclusive rights to pursue development, registration and commercialization in all countries outside the U.S.

Upon the transfer of the marketing authorization from Spark Therapeutics to Novartis, Novartis can commercialize Luxturna in the EU/European Economic Area (EEA). Spark Therapeutics entered into a separate agreement with Novartis to manufacture and supply Luxturna to Novartis.

“The historic approval of Luxturna in Europe furthers our mission to challenge the inevitability of genetic disease around the world. Following the launch of Luxturna in the United States earlier this year, this decision makes Luxturna the first gene therapy for a genetic disease approved in both the U.S. and EU, a promising milestone for the many people living with genetic disease around the world,” said Ron Philip, a chief commercial officer at Spark Therapeutics. “Having worked closely with the global IRD community on the development of Luxturna, we are proud to be able to share the first gene therapy treatment option for an inherited retinal disease (IRD) with appropriate patients in Europe, in collaboration with Novartis.”

In September 2018, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending approval of Luxturna. This decision was 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 in both copies of the RPE65 gene. The Phase 3 trial was the first randomized, controlled gene therapy trial for a genetic disease. Luxturna is designated as an orphan medicinal product for the treatment of inherited retinal dystrophies.

Luxturna was approved by the U.S. Food & Drug Administration (FDA) in December 2017.

Luxturna is first gene therapy for a genetic disease approved in both the U.S. and EU. Luxturna is first and only gene therapy approved in EU for patients with an inherited retinal disease (IRD).