Spark's Luxturna Approval Ushers In A New Gene Therapy Era

Spark Therapeutics Inc. has earned a place in the pharma history books on two fronts, as the first drug maker to secure a gene therapy approval from FDA and the first company to develop a treatment for blindness. But it remains to be seen whether it reaches another first – a $1m price tag.

Luxturna (voretigene neparvovec-rzyl) was approved by FDA with a broad label Dec. 19 as a one-time gene therapy for patients with biallelic RPE65 mutation-associated retinal dystrophy. It's worth pausing to reflect on the accomplishment: manipulating an individual's genes to relieve a debilitating form of blindness