1. Jul 15, 2021

FDA grants breakthrough therapy designation for ALK-001 for Stargardt Disease

The U.S. Food and Drug Administration has granted breakthrough therapy designation to ALK-001 (C20-D3-vitamin A; Alkeus Pharmaceuticals) for the treatment of Stargardt Disease, according to a press release.

ALK-001 is a chemically modified form of vitamin A that has been shown to slow the progression of Stargardt while preserving the normal visual cycle. This is the first drug to this designation for Stargardt Disease.

“Obtaining Breakthrough Therapy Designation is a transformative milestone in Stargardt disease,” Leonide Saad, PhD, CEO of Alkeus Pharmaceuticals, said in a company news release. “The results from our phase 2 trial provide a strong basis for regulatory filing and approval of ALK-001 for the treatment of Stargardt disease. We look forward to working with the FDA and other regulatory agencies so that we can bring ALK-001 to patients as quickly as possible.”

Read the full press release here.