• linkedin
  • Increase Font
  • Sharebar

    FDA approval of retinal dystrophy drug launches era of ocular gene therapy

    By Cheryl Guttman Krader; Reviewed by Alex V. Levin, MD, and Paulo Falabella, MD

    In December 2017, the FDA approved voretigene neparvovec-rzyl (“voretigene,”  Luxturna, Spark Therapeutics), an adeno-associated virus serotype 2 vector-based gene therapy, for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy (RD).

    With the regulatory agency’s decision, Luxturna became the first pharmacologic treatment for this inherited, progressive disease that often leads to nearly complete blindness and the first gene therapy in the United States indicated for treatment of a genetic disease. The treatment is expected to be available in March 2018.

    Its approval was granted based on clinical trial results showing that the gene therapy had an acceptable safety profile and resulted in rapid improvements in functional vision and visual function that were sustained with follow-up to 2 years. Data reported at the 2017 American Academy of Ophthalmology meeting show the treatment benefit persists to at least 3 years.

    “The approval heralds a new horizon for treatment of inherited RDs,” said Alex V. Levin, MD, MHSc, chief, pediatric ophthalmology and ocular genetics, Wills Eye Hospital, Philadelphia. “It behooves affected patients to get accurate molecular diagnosis of their underlying mutations so that they can bring themselves closer to intervention.”

    “FDA approval of Luxturna is a landmark event that represents the culmination of nearly 20 years of hard work and a breakthrough for patients affected with biallelic RPE65 mutation-associated RD, their families, and the scientific community,” added Paulo Falabella, MD, medical affairs ophthalmic lead, Spark Therapeutics, Philadelphia. Needless to say, Spark Therapeutics is very pleased and excited to bring it to market.”


    Cheryl Guttman Krader
    Cheryl Guttman is a medical writer based in Deerfield, Ill.


    You must be signed in to leave a comment. Registering is fast and free!

    All comments must follow the ModernMedicine Network community rules and terms of use, and will be moderated. ModernMedicine reserves the right to use the comments we receive, in whole or in part,in any medium. See also the Terms of Use, Privacy Policy and Community FAQ.

    • No comments available

    Latest Tweets Follow

    var script= ' ';