Shares of Spark Therapeutics Inc. (NASDAQ: ONCE) saw a handy gain to close out the week after the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee unanimously recommended approval of Luxturna (voretigene neparvovec).
Specifically, Luxturna is being recommended for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD).
The recommendation was ultimately based on Luxturna’s clinical development program, which includes the first completed controlled Phase 3 gene therapy clinical trial ever conducted for a genetic disease. In the original Phase 3 intervention group, participants aged four to 44 years on average maintained the functional vision and visual function improvements demonstrated 30 days after Luxturna administration through their last annual follow-up visit.
No serious adverse events (SAEs) associated with Luxturna or deleterious immune responses have been observed. Two ocular SAEs were reported in the clinical program, one in Phase 1 and one in Phase 3. The SAE in Phase 3 was related to the surgical procedure.
Luxturna is under Priority Review with the FDA, with an assigned Prescription Drug User Fee Act (PDUFA) date of January 12, 2018.
Katherine A. High, M.D., president and head of Research and Development, Spark Therapeutics, commented:
Today’s unanimous advisory committee vote recommending the approval of Luxturna moves us closer to bringing this investigational adeno-associated viral (AAV) vector gene therapy to patients with vision loss due to confirmed biallelic RPE65-mediated IRD. The clinical program for Luxturna includes patient data that show efficacy for up to four years on endpoints including bilateral multi-luminance mobility test (MLMT) score change and full-field light sensitivity threshold (FST) testing, with observation ongoing. We look forward to continuing to work with FDA as it completes its review of Luxturna.
Shares of Spark were about 3% at $88.55 early Friday, with a consensus analyst price target of $92.56 and a 52-week range of $35.07 to $91.00.