A US Food and Drug Administration (FDA) advisory committee recommended that voretigene neparvovec be approved to treat biallelic RPE65-mediated inherited retinal dystrophy (IRD) last week, basing the decision on Phase III data showing it improved vision in 93% of recipients.
The therapy – which has been given the provisional name Luxturna - is a recombinant adeno-associated viral (AAV) vector serotype 2, expressing the gene for human retinal pigment epithelial 65 kilodalton protein (AAV2-hRPE65v2).
The Food and Drug Administration (FDA) is due to make a final decision in January.
Patient groups and financial analysts welcomed the recommendation.
The Alliance for Regenerative Medicine (ARM), a US advocacy group that lobbies on behalf of advanced therapy manufacturers, was also positive about the endorsement.
A spokeswoman told us "ARM believes this endorsement serves as an acknowledgement of the profound potential of gene therapy."
"This, combined with the FDA’s recent approval of Novartis’s cell-based immunotherapy CAR-T product, Kymriah, and the agency’s implementation of the new RMAT designation for cell and gene therapies, is additional evidence of the growing awareness and validation of this sector and the significant patient benefits."
According to ARM, at present 504 gene therapies are in clinical development around the world, 34 of which are in Phase III clinical trials.
Spark has not said how much it will charge for Luxturna. According to analysts, the single-shot treatment may cost as much as $1m.
According to a document Spark filed with the Securities Exchange Commission (SEC) in February, “there are approximately 3,500 individuals with RPE65 -mediated IRD in the United States, as well as France, Germany, Italy, Spain and the United Kingdom.”