Stargardt disease (or fundus flavimaculatus) is the most common form of inherited macular degeneration, with a prevalence of about 1 per 10,000 births. The disease causes uncorrectable loss of central vision, with most patients experiencing initial symptoms prior to age 20.
Stargardt disease has been shown to be the result of a mutation in the ABCA4 (or ABCR) gene. This mutation leads to production of a dysfunctional Rim protein, which is responsible for the intracellular transport of vitamin A. As a result, vitamin A accumulates and forms toxic aggregates within the photoreceptors, leading to eventual vision loss. SAR 422459 injections deliver a healthy version of the ABCR gene into the retina, allowing proper production of the Rim protein to clear these aggregates.
Sanofi is actively enrolling patients in subretinally injected SAR 422459 Phase IIa clinical trials for Stargardt Disease. There is currently no cure or treatment for Stargardt disease, but competitors are developing alternatives to SAR 422459 such as ALK-001 by Alkeus and MA09-hRPE by Astellas Pharma and Ocata Therapeutics.
Managed Care Segment
Selected Sample of Epidemiology Sources Used in This Analysis
Chacón-camacho OF, Granillo-alvarez M, Ayala-ramírez R, Zenteno JC. ABCA4 mutational spectrum in Mexican patients with Stargardt disease: Identification of 12 novel mutations and evidence of a founder effect for the common p.A1773V mutation. Exp Eye Res. 2013;109:77-82.
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