FDA Panel Votes Unanimously in Favor of Gene Therapy to Treat Rare Eye Disorder

Philadelphia-based Spark Therapeutics took its gene therapy before the US Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee on Thursday and the panel voted 16 to 0 in favor of approving the subretinal injection, representing a major step forward for adeno-associated virus gene therapies.

The therapy, known as Luxturna (voretigene neparvovec), has been granted priority review by FDA (the agency has until 12 January 2018 to make its decision) as a one-time treatment for patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD).

At the committee’s outset, Wilson Bryan, director of FDA’s Office of Tissues and Advanced Therapies, raised questions about Spark’s use of a novel endpoint and whether meeting it represents a true improvement in the lives of these patients.

But the data and patient testimonies outweighed any question marks. The company pointed to the improved vision in more than 90% of clinical trial participants based on their ability to navigate an obstacle course in low light, though that percentage fell to 72% a year after treatment.

The clinical trial program included 41 participants with vision loss aged four to 44 at the time of first administration and several of the younger patients discussed their participation in the trial on Thursday, noting the ways in which the treatment helped them to regain eyesight and improve their lives.

Biotech analysts seem largely in agreement that the therapy will win FDA approval, though like with the approval in August of Novartis’ CAR-T therapy Kymriah (tisagenlecleucel), which came with a $475,000 price tag, questions will be raised over the price tag of Spark’s gene therapy.

Although there is only a market of about 6,000 eligible patients worldwide, Leerink analysts have said Spark’s treatment could cost $600,000 per patient in the US and $400,000 in the EU.

2017 Meeting Materials, Cellular, Tissue and Gene Therapies Advisory Committee