FDA Committee recommends gene therapy for retinal disease to help prevent blindness

Frank
May 19, 2018
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In an encouraging sign for Spark Therapeutics, a U.S. Food and Drug Administration Committee voted unanimously to recommend approval of its new biologics application for Luxturna a gene therapy to treat patients with vision loss due to an inherited retinal disease, according to a company news release. Such favorable committee votes frequently bode well for FDA approval.

The FDA Cellular, Tissue and Gene Therapies Advisory Committee voted unanimously to recommend the treatment.

There is currently no known treatment for RPE65-mediated inherited retinal disease, which usually leads to complete blindness, according to principal Investigator Dr. Albert Maguire, who is also a professor of ophthalmology at the Scheie Eye Institute at the University of Pennsylvania’s Perelman School of Medicine. He is also a physician in the Division of Pediatric Ophthalmology at Children’s Hospital of Philadelphia.

A Phase 3 trial of Luxturna was conducted from 2013 to 2015. The clinical trial had 41 participants aged four to 44 years old with vision loss spanning from mild to advanced. Confirmed biallelic RPE65 mutations and the presence of sufficient viable retinal cells were established in all participants, according to the release.

Still, the positive news was tempered with the reality that the effectiveness of the treatment vary depending on the number of viable retinal cells patients have left when they receive the treatment, Endpoints noted citing FDA documents.

Other critical questions remain, namely the price this gene therapy would command and how Spark Therapeutics would charge for it.

The gene therapy developer is a spin out of Children’s Hospital of Philadelphia.

The FDA set a date of January 12 next year to review whether to grant approval for Luxturna.

Other products in Spark’s pipeline include gene therapy programs to address neurodegenerative diseases, hematologic disorders and other forms of inherited blindness.

Photo: Getty Images

Frank’s source: https://medcitynews.com/2017/10/fda-committee-recommends-gene-therapy-retinal-disease-help-prevent-blindness/

 

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