FDA Approves Novel Gene Therapy to Treat a Rare Form of Inherited Vision Loss

Source: Spark Therapeutics

Summary: The US-FDA approved a novel gene therapy – Luxturna, first of its kind to treat children and adult patients with a rare inherited form of vision loss that may result in blindness.

Spark Therapeutics at Philadelphia is a fully integrated late clinical-stage gene therapy company. Its investigational therapies have the potential to provide long-lasting effects, dramatically and positively changing the lives of patients with conditions where no, or only palliative, therapies exist. Greater understanding of the human genome and genetic abnormalities have allowed its scientists to tailor investigational therapies to patients suffering from very specific genetic diseases.

The US-FDA (Food and Drug Administration) has approved LUXTURNA™ (voretigene neparvovec-rzyl), a one-time gene therapy product for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. LUXTURNA is the first FDA-approved gene therapy for a genetic disease, the first and only pharmacologic treatment for an inherited retinal disease (IRD) and the first adeno-associated virus (AAV) vector gene therapy approved in the U.S.

Gene therapy to treat a rare inherited vision loss

LUXTURNA (voretigene neparvovec-rzyl) product vial (green cap) and two diluent solution vials (white caps), used to dilute the product. Credit: Spark Therapeutics, Inc

Luxturna should be administered to only those patients with mutations on both copies of the RPE65 gene who have sufficient viable retinal cells. Treatment with Luxturna must be done separately in each eye on separate days, with at least six days between surgical procedures. It is administered via subretinal injection by a surgeon experienced in performing intraocular surgery. A short course of oral prednisone treatment is given to the patients to limit the potential immune reaction to Luxturna

The safety and efficacy of Luxturna were established in a clinical development program with a total of 41 patients between the ages of 4 and 44 years. All participants had confirmed biallelic RPE65 mutations. The group of patients that received Luxturna demonstrated significant improvements in their ability to complete the obstacle course at low light levels as compared to the control group. The most common adverse reactions from treatment with Luxturna included eye redness (conjunctival hyperemia), cataract, increased intraocular pressure and retinal tear.

Jeffrey D. Marrazzo, a chief executive officer at Spark Therapeutics said, “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. We offer our sincere gratitude to the patients and their families as well as the expert investigators who continue to participate in LUXTURNA’s clinical development program.”

More Information: “FDA Approves Spark Therapeutics’ LUXTURNA™ (voretigene neparvovec-rzyl), a One-time Gene Therapy for Patients with Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy”.


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