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FDA panel supports Spark Therapeutics gene therapy approval for rare form of blindness

PBR Staff Writer Published 13 October 2017

Spark Therapeutics’ gene therapy Luxturna (voretigene neparvovec) has been recommended for approval by an advisory panel of the US Food and Drug Administration (FDA) for the treatment of blindness caused by an inherited retinal disease (IRD).

The investigational adeno-associated viral (AAV) vector gene therapy was unanimously approved by all the 16 members of the Cellular, Tissue and Gene Therapies Advisory Committee of the drug regulator.

Luxturna, a one-time gene therapy is now a step closer from bagging the FDA approval to treat patients who lost their vision due to confirmed biallelic RPE65-mediated IRD.

The committee’s recommendation comes on the back of the results of a phase 3 gene therapy clinical trial of Luxturna.

Through the trial, Spark Therapeutics established that Luxturna was successful in maintaining the functional vision and improving visual functions of the participating patients, 30 days after its administration.

The gene therapy was found to have increased the ability of 93% of the patients in navigating obstacles in low light conditions.

Spark Therapeutics research and development president and head Katherine High said: “The clinical program for LUXTURNA includes patient data that show efficacy for up to four years on endpoints including bilateral multi-luminance mobility test (MLMT) score change and full-field light sensitivity threshold (FST) testing, with observation ongoing.”

RPE65-mediated inherited retinal disease is a rare form of vision loss to which there are no pharmacologic treatment options available as of now. Patients with this disease often face night blindness (nyctalopia) and in a majority of cases, the form of IRD leads to complete blindness.

Luxturna is intended to be given only once to the patients, intravenously (IV) or injected into specific tissue. The gene therapy delivers viral vector particles having the correct copy of the RP65 gene to retinal cells, thereby repairing their ability to produce the deficient enzyme.


Spark Therapeutics research and development president and head Katherine High. Photo: courtesy of Spark Therapeutics, Inc.