Spark Therapeutics shares were higher over 3% on Monday after the company said the U.S. Food and Drug Administration granted a request to amend the orphan drug designation for voretigene neparvovec to the treatment of inherited retinal dystrophy due to biallelic RPE65 mutations.
The company said the expanded designation aligns with the proposed indication for voretigene neparvovec.
Meanwhile, the company said four-year data from a phase 1 clinical trial and natural history study enhance the understanding of investigational voretigene neparvovec.
“Beyond an initial gain of functional vision seen in 93 percent of all phase 3 trial participants at year one, these four-year data from the phase 1 cohort further add to the clinical evidence relating to the potential long-term effect of one administration of investigational voretigene neparvovec on IRD caused by biallelic RPE65 mutations,” CEO Jeffrey Marrazzo said.
By Stephen Holmes