Gene therapy continues to advance

FDA’s Advisory Committee unanimously recommends that Luxturna from the American gene therapy company Spark Therapeutics will be approved for the treatment of an unusual form of eye disease called retinitis pigmentosa

Retinitis pigmentosa is a genetic disease. Symptoms comprise of, among other things, involuntary eye movements, tunnel vision and loss of night vision. The disease can lead to complete blindness. No effective treatment has previously existed, but the recommendation from the FDA’s Advisory Committee probably means that the FDA (the Federal Drug Administration) will approve Luxturna for use on the American market. If Luxturna obtains final approval from the FDA, it will be the first approved gene therapy treatment for a genetic disease in the USA. The treatment can potentially be administered once and have a lifetime effect on the patient.

Luxturna has carried out four years of clinical studies. No gene therapy-related side-effects have been identified. A few side-effects have arisen in connection with the surgical operation through which the gene therapy is administered. The recommendation from FDA’s Advisory Committee to approve Luxturna is another great success for gene therapy of late. In August 2017, the FDA approved Kymriah from the pharmaceutical company Novartis for the treatment of certain paediatric patients and young people with a treatment-resistant type of acute lymphatic leukaemia.

To Sparks pressrelease:

The National Board of Health and Welfare (Socialstyrelsen):