FDA approves Vitamin A as breakthrough therapy for Stargardt disease and AMD
ALK-001 is a chemically-modified vitamin A which has been granted a breakthrough by the Food and Drug Administration (FDA) for the prevention and treatment of Stargardt disease and age-related macular degeneration (AMD).
Stargardt disease is the most common inherited macular dystrophy affecting both adults and children, and having a prevalence of 1 in 8000–10 000. STGD1 has an autosomal recessive mode of inheritance associated with disease-causing mutations in the ABCA4 gene. It is both clinically and genetically highly heterogeneous.
However, toxic vitamin A aggregates ("dimers") have been implicated in the pathophysiology of Stargardt and dry-AMD. Vitamin A dimers form when two molecules of vitamin A are combined. Specifically, ALK-001 is vitamin A modified by replacing hydrogen with deuterium, a stable, non-radioactive isotope of hydrogen. Substituting vitamin A with ALK-001 safely slows the rate of formation of vitamin A dimers and prevents the disease in mice.
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