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Spark Therapeutics announces additional efficacy outcomes from first randomised US trial in gene therapy

Spark Therapeutics (NASDAQ:ONCE), a gene therapy company based in Philadelphia, USA, has announced announced that the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee has unanimously recommended (16-0) approval of “LUXTURNA” (voretigene neparvovec), an investigational, “potential one-time gene therapy, for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD).” Somewhere between one and two thousand patients in the US are understood to suffer from the disorder.


Commenting on the milestone, Katherine A. High, M.D., President and Head of Research and Development at Spark Therapeutics stated, the “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.”


Approval of LUXTURNA, previously “SPK-RPE65” was secured on the company’s phase III clinical trial data, which included data from an intent-to-treat population of patients randomized over a 12-month time period. According to the company, the clinical trial results showed “a statistically significant and clinically meaningful difference between intervention (n=21) and control participants (n=10) at one year, per the clinical trial’s primary endpoint, mean bilateral multi-luminance mobility testing (MLMT) score change (difference of 1.6; 95% CI, 0.72, 2.41; p=0.001).” The company additionally announced that treated patients had a “marked difference compared to control participants across the first two secondary endpoints: full-field light sensitivity threshold (FST) testing averaged over both eyes (p=0.001) and the mobility test score change for the first injected eye (p=0.001).”