Rare Daily Staff

The U.S. Food and Drug Administration granted Breakthrough Therapy designation to Alkeus Pharmaceuticals’ experimental therapy ALK-001 for the treatment of 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.

ALK-001 is a chemically-modified form of vitamin A developed to treat multiple retinal degenerative diseases. Clinical data indicates that ALK-001 safely slows the progression of Stargardt while preserving the normal visual cycle.

“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,” said Leonide Saad, CEO of Alkeus Pharmaceuticals.

Breakthrough Therapy designation is a process designed by the FDA to expedite the development and review of promising experimental drugs. Previously, the FDA granted ALK-001 Orphan Drug Designation.

The FDA granted ALK-001 Breakthrough Therapy Designation after reviewing data from Alkeus Pharmaceuticals’ phase 2, double-masked, randomized, placebo-controlled trial in Stargardt disease. Data from the trial and other ongoing studies are expected to be reported later in the year.

Clinical trials for ALK-001 are also underway for patients with dry age-related macular degeneration (AMD). AMD is the number one cause of unpreventable blindness in the USA.

Photo: Leonide Saad, CEO of Alkeus Pharmaceuticals

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