Orphan Drug Designation for MI-100 in Stargardt's Disease
Makindus announced that it has received orphan drug designation from the FDA for MI-100 for the treatment of Stargardt's Disease.
Stargardt's, also known as juvenile macular degeneration, is a genetic disease caused by mutations in the ABCA4 gene. Disease onset is usually around 8–12 years old with significant variability in the speed of disease progression. Makindus plans to initiate a Phase 3 confirmatory trial early 2015 and may begin enrolling patients as early as March 2015.
For more information call (267) 291-4912 or visit Makindus.com.