FDA Grants Breakthrough Therapy Designation To Spark Therapeutics' SPK-RPE65 For Retinal Dystrophies

By Cyndi Root

Spark Therapeutics announced in a press release that the Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to its lead candidate, SPK-RPE65. The designation signifies that the therapy shows advantages over existing therapies and is intending to treat a serious or life-threatening condition. SPK-RPE65 received the status for nyctalopia (night blindness), in patients with Leber's congenital amaurosis due to mutations in the RPE65 gene, however Spark intends to develop the agent for group of blinding conditions known as inherited retinal dystrophies (IRDs). Jeffrey D. Marrazzo, co-founder and CEO of Spark Therapeutics, said, "FDA's breakthrough therapy designation for SPK-RPE65 underscores the serious unmet medical needs faced by patients with genetic blinding conditions.”

SPK-RPE65

SPK-RPE65 targets the mutations in the RPE65 gene that lead to IRDs and visual impairments. Expressed in the retinal pigment epithelium (RPE), the RPE65 gene codes the protein that converts light into an electrical signal that the brain interprets as sight. Spark’s agent works with a neutralized vector virus and delivers the RPE65 gene into the eye, producing new genetic material and the protein needed to improve sight.

In two early trials of SPK-RPE65, IRD patients showed improved capabilities in daily living and independent activities. Patients were followed long-term and the effects of the agent seem long lasting. For instance, SPK-RPE65-treated children saw increased mobility. Currently, Sparks is conducting a fully enrolled Phase 3 trial that seeks to restore functional vision in patients, and the company intends to report data from that trial in the second half of 2015. It also intends to file a Biologics License Application (BLA) with the FDA in 2016 for SPK-RPE65.

Spark Activities

In the same press release announcing SPK-RPE65’s breakthrough status, Spark also gave an update on its activities. The company made several appointments in senior leadership including Katherine High, M.D., as its president and CSO.

Sparks also announced that it had established new headquarters in Philadelphia, which it expects to be complete by the end of the year.