US FDA approves Spark’s gene therapy Luxturna for rare form of vision loss

Luxturna FDA approval : Luxturna (voretigene neparvovec), a gene therapy from Spark Therapeutics has secured the approval from the US Food and Drug Administration (FDA) for a rare inherited type of vision loss affecting children and adults that could result in blindness if left untreated.

Luxturna is an adeno-associated viral (AAV) vector gene therapy which is indicated to be given as a one-time gene therapy in patients having Biallelic RPE65 mutation-associated retinal dystrophy.

Such patients are often troubled with night blindness (nyctalopia). In most of these patients, the RPE65-mediated inherited retinal disease can lead to complete vision loss.

The findings of a phase 3 trial held across 34 patients having confirmed biallelic RPE65 mutations led to Luxturna FDA approval. In this phase 3 trial, the enrolled patients after 30 days of receiving Luxturna gene therapy could sustain their functional vision apart from experiencing better visual functions.

Spark Therapeutics Luxturna gene therapy
Spark Therapeutics Luxturna gene therapy. Photo courtesy of Spark Therapeutics, Inc.

The phase 3 trial also demonstrated that Luxturna gene therapy increased the ability of the patients in overcoming obstacles in poor light conditions by an overwhelming margin of 93%.

Commenting on Luxturna FDA approval, Jeffrey Marrazzo – CEO of  Spark Therapeutics CEO Jeffrey Marrazzo said: “Today’s landmark approval of LUXTURNA is a moment decades in the making for the field of gene therapy, the inherited retinal disease (IRD) community, and most importantly, patients with biallelic RPE65 mutation associated retinal dystrophy who now have the option to seek treatment.

“This one-time gene therapy for an inherited disease represents a first-of-its-kind breakthrough that may lay the groundwork for the development of gene therapies for other conditions that are not adequately addressed today.”

With its approval, Luxturna is the first ever pharmacologic treatment available for an inherited retinal disease (IRD). It is also the first adeno-associated viral to get the nod of the FDA.

Luxturna gene therapy should be administered only to patients having mutations on the two copies of the RPE65 gene and those who have adequate viable retinal cells as confirmed by their treating physicians.

The gene therapy has to be given intravenously or injected into specific tissue. Luxturna works by releasing viral vector particles having the correct version of the RP65 gene to retinal cells. As a result, the gene therapy helps in repairing the ability of the retinal cells to produce the deficient enzyme.

Commenting on Luxturna FDA approval, Scott Gottlieb – Commissioner of FDA, said: “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.”

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