World's First Gene Therapy For Inherited Blindness Gets EU Approval
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World’s First Gene Therapy For Inherited Blindness Gets EU Approval

In a landmark achievement, Novartis has received consent for Luxturna, a gene treatment that may restore vision in people who have a particular genetic mutation which causes progressive vision loss. The treatment is suggested for people with mutations at the two copies of a gene, called RPE65, that cause retinal cells to die over time, leading to progressive loss of vision which begins in childhood. There is no treatment for people with this illness. The ones that have functioning cells and still have a result in a genetic evaluation can be treated with the gene treatment.

In a Phase III trial, one dose of this treatment improved the vision of patients as measured via a test where patients need to locate their way. Improvements were regarded as early as 30 days following the treatment. Developed by US-based Spark Therapeutics, the therapy was approved almost a year back in the US, where its cost is the most expensive for any drug on the planet at $750,000 ($850,000). Novartis is now in the process of negotiating settlement and pricing.

“The very first national reimbursement approvals of Luxturna are anticipated in 2019,” Dirk Sauer, Global Development Unit Head of Novartis Ophthalmology, said.”The timelines for the reimbursement process in France, Germany and countries from the Nordics suggest that these might be the very first countries where patients could be able to access Luxturna, with quite a few other countries after.”

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“EU approval of the one-time gene therapy Luxturna marks a milestone in reimagining World's First Gene Therapy For Inherited Blindness Gets EU Approvalmedicine and can bring real value to patients, their families, and society as a whole,” said Paul Hudson, CEO, Novartis Pharmaceuticals. “Novartis is committed to working with patients, caregivers, health 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 treatment options.”

Many others are in development, although luxturna is your first gene therapy for a hereditary type of blindness. In Europe, companies including GenSight, Nightstar and Horama are developing their own gene therapy approaches for different kinds of genetic blindness.

Source: Novartis

Image courtesy: Novartis

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