The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to ALK-001 (C20-D3-vitamin A) for the treatment of Stargardt disease.

Stargardt disease is a rare, progressive inherited retinal degenerative disease that typically begins in childhood or adolescence causing irreversible vision loss leading to blindness. The disease is most commonly caused by mutations in the ABCA4 gene, which results in the accumulation of toxic vitamin A aggregates. The Company believes these vitamin A dimers are partially responsible for vision loss.

ALK-001 is an oral, once daily, chemically-modified form of vitamin A designed to reduce the production of toxic vitamin A dimers by substituting vitamin A and retaining its natural functions. In preclinical trials, ALK-001 has been shown to slow the formation of vitamin A dimers and prevent the disease in mice.

The Breakthrough Therapy designation is based on data from the double-masked, randomized, placebo-controlled phase 2 TEASE trial ( Identifier: NCT02402660), which assessed the safety and tolerability of ALK-001, as well as its effects on the progression of Stargardt disease. Results showed that treatment with ALK-001 demonstrated statistically and clinically significant efficacy. Study data will be presented and published later this year.

“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, PhD, CEO of Alkeus Pharmaceuticals. “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.”

The Company is also investigating ALK-001 for the treatment of geographic atrophy secondary to dry age-related macular degeneration in a phase 3 study ( Identifier: NCT03845582).


  1. FDA grants Alkeus Pharmaceuticals Breakthrough Therapy designation for ALK-001 (C20-D3-vitamin A) for the treatment of Stargardt disease. [press release]. Somerville, MA: Alkeus Pharmaceuticals, Inc.; July 14, 2021. 
  2. Science: ALK-001. Alkeus Pharmaceuticals website. Accessed July 14, 2021.