FDA Grants Breakthrough Therapy Designation for Stargardt Disease Treatment

Alkeus Pharmaceuticals, Inc., a private, late-stage biopharmaceutical company, announced that the U.S. FDA granted Breakthrough Therapy Designation to ALK-001 for the treatment of Stargardt Disease.

ALK-001 (C20-D3-vitamin A) is a chemically modified form of vitamin A developed to treat multiple retinal degenerative diseases. The investigational treatment is taken once a day. Clinical data indicates that ALK-001 safely slows the progression of Stargardt while preserving the normal visual cycle. ALK-001 is the only drug to receive Breakthrough Therapy Designation for Stargardt Disease.

Stargardt disease is a progressive inherited retinal degenerative disease that causes irreversible vision loss leading to blindness. An estimated 40 to 60,000 people in the United States have this rare and serious condition. Symptoms typically begin in childhood or adolescence. There is currently no approved therapy for Stargardt disease. Almost everyone diagnosed with the disease will become legally blind.

“Obtaining Breakthrough Therapy Designation is a transformative milestone in Stargardt disease,” said Leonide Saad, PhD, CEO of Alkeus Pharmaceuticals. “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.”