Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating genetic diseases, has announced that its lead product candidate, SPK-RPE65, has received Breakthrough Therapy designation from the FDA. Breakthrough Therapy designation is intended to expedite the development and review of investigational therapies for serious or life-threatening conditions where initial clinical evidence demonstrates a significant improvement over existing therapies. SPK-RPE65 targets a group of blinding conditions known as inherited retinal dystrophies (IRDs) caused by autosomal recessive mutations in the RPE65 gene, and for which there currently is no pharmacologic treatment available.
"FDA's Breakthrough Therapy designation for SPK-RPE65 underscores the serious unmet medical needs faced by patients with genetic blinding conditions," said Jeffrey D. Marrazzo, co-founder and CEO, Spark Therapeutics.
SPK-RPE65 received Breakthrough Therapy designation for the treatment of nyctalopia, or night blindness, in patients with Leber's congenital amaurosis due to mutations in the RPE65 gene. The designation was supported by two earlier clinical trials where results to date and reports from the clinical study team suggest that SPK-RPE65 enables patients to perform activities of daily living with greater independence than prior to treatment, and has long-lasting effects in restoring functional vision.
Notably, as reported by the clinical study team, following a single injection of SPK-RPE65 in one eye, children in the trials no longer depended on visual aids to carry out classroom activities and were able to walk and play more like normally sighted children.
SPK-RPE65 currently is in a fully-enrolled pivotal phase III clinical trial. Spark expects to report data from this phase III trial in the second half of 2015.
The receipt of Breakthrough Therapy designation for SPK-RPE65 follows several recent and significant corporate developments for Spark as it moves toward completing clinical development of SPK-RPE65: