Published On: Sat, Nov 24th, 2018

Spark Therapeutics bags Luxturna EU approval for inherited retinal disease

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Luxturna EU approval for : Pennsylvania-based Spark Therapeutics has bagged the European Commission’s (EC) approval for its gene therapy Luxturna (voretigene neparvovec) to treat a rare inherited type of vision loss in both children and adults.

Luxturna EU approval for inherited

Luxturna, an adeno-associated viral (AAV) vector gene therapy has been indicated to be given as a one-time gene therapy in patients with biallelic RPE65 mutation-associated inherited retinal dystrophy. Luxturna EU approval is for such patients who have enough viable retinal cells and is applicable in all the 28 member states and also Norway, Iceland and Liechtenstein.

It can be recalled that the Spark Therapeutics gene therapy bagged approval from the US in December 2017 for the same indication. Luxturna FDA approval can be read here.

In January 2018, Swiss pharma giant acquired the ex-US licensing rights for Luxturna for $170 million under which it will market the gene therapy in Europe and rest of the world.

Luxturna EU approval for inherited retinal disease

Spark Therapeutics announces Luxturna EU approval for inherited retinal disease. Photo courtesy of Spark Therapeutics, Inc.

Commenting on Luxturna EU approval for inherited retinal disease, Paul Hudson – CEO of Novartis Pharmaceuticals said: “EU approval of the one-time gene therapy Luxturna marks a milestone in reimagining medicine and can bring real value to patients, their families and society as a whole.

“Novartis is committed to working with patients, caregivers, systems and physicians to establish access to this gene therapy for RPE65 patients, as we believe it can help restore sight and improve vision in children and adults who currently have no options.”

Luxturna EU approval for inherited retinal disease means that it is now the first gene therapy to be approved in the US and EU. It is also the first and only gene therapy to get an EU approval for patients suffering from an inherited retinal disease.

Ron Philip, chief commercial officer at Spark Therapeutics, commenting on Luxturna EU approval for inherited retinal disease, said: “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.”

Luxturna EU approval was driven by the positive opinion taken by the European Medicines Agency’s Committee for Medicinal Products for Human Use in September.

Overall, the approval of the Spark Therapeutics gene therapy came on the basis of the findings of three clinical trials that featured a of 43 patients with inherited retinal disease. It included a phase 1 trial, a follow-up clinical trial of that and a phase 3 clinical trial.

Luxturna Mechanism of Action

Luxturna mechanism of action is by the release of viral vector particles having the correct version of the RP65 gene to retinal cells. Owing to this, the gene therapy aids in restoring the ability of the retinal cells to generate the deficient enzyme.

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