FDA Approves Spark Therapeutics’ Gene Therapy for Inherited Vision Loss

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FDA has approved a new gene therapy for treating patients born with a rare, inherited vision loss.

On Dec. 19, 2017, FDA approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy developed by Spark Therapeutics, a Philadelphia, PA-based late clinical-stage gene therapy company. The product is approved for treating an inherited form of vision loss that may result in blindness and is the first directly administered gene therapy approved in the United States that targets a disease caused by mutations in a specific gene, according to FDA.

Specifically, Luxturna is approved to treat patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy that leads to vision loss and may cause complete blindness in certain patients. FDA granted priority review, breakthrough therapy, and orphan drug designations to the product’s application.

Luxturna will be manufactured at Spark Therapeutics’ manufacturing facility in West Philadelphia. According to the company, this facility is the first licensed manufacturing facility in the US for a gene therapy treating an inherited disease. The therapy will be administered at selected treatment centers in the US and is expected to be available late in the first quarter of 2018.

“Today’s approval marks another first in the field of gene therapy-both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss-and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases. The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious and rare diseases. I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses,” said FDA Commissioner Scott Gottlieb, MD, in an agency press release.


“We’re at a turning point when it comes to this novel form of therapy and at [FDA], we’re focused on establishing the right policy framework to capitalize on this scientific opening. Next year, we’ll begin issuing a suite of disease-specific guidance documents on the development of specific gene therapy products to lay out modern and more efficient parameters-including new clinical measures-for the evaluation and review of gene therapy for different high-priority diseases where the platform is being targeted,” Gottlieb said in the press release.

Source: FDA and Spark Therapeutics