Spark Therapeutics’ vision-restoring RPE65 gene therapy has received marketing approval from the U.S. Food and Drug Administration, becoming the first gene therapy to gain regulatory approval in the U.S. for the eye or any inherited condition.
Today’s U.S. Food and Drug Administration (FDA) approval of voretigene neparvovec, to be marketed as LUXTURNA, will be life-changing for patients with vision loss due to mutations in the RPE65 gene and a watershed moment for the inherited retinal disease field, says the Foundation Fighting Blindness.
The Foundation Fighting Blindness (FFB), the world’s leading private funder of research on treatments and cures for vision-robbing inherited retinal diseases, strongly opposes recent U.S. Senate and House of Representatives actions which would slow or stop research on treatments for Americans impacted by rare diseases.
Though the U.S. Food & Drug Administration (FDA) is more tightly regulating U.S. clinics that tout stem-cell trials and treatments for inherited retinal diseases (IRDs) and a host of other conditions, many significant loopholes and enforcement limitations remain.
ProQR, a biotech company in the Netherlands, has treated its first patient in a Phase 1/2 clinical trial for QR-110, a therapy for people with Leber congenital amaurosis 10 (LCA10), which is caused by the p.Cys998X mutation in the CEP290 gene. The mutation is estimated to affect about 2,000 people in the Western world.
The Foundation Fighting Blindness (FFB) announced that it has selected Benjamin Yerxa, a biotechnology and drug development executive, as its Chief Executive Officer, effective immediately.
Ashley and Cole Carper traveled from Little Rock, AR, to tell their family’s story at the FDA hearing.
Approval of the gene therapy will allow treatment for people with vision-loss due to RPE65 mutations and will help advance other investigational studies with potential to help millions, FFB tells the Committee.
With five gene-therapy clinical trials underway or soon to begin, Applied Genetic Technologies Corporation (AGTC) is generating tremendous excitement for the potential to overcome vision loss from several inherited retinal diseases.