In a key catalyst for the small biotech, Modus Therapeutics AB has won rare pediatric disease designation from the FDA, raising the Swedish group's chances of winning a coveted rare disease priority review voucher if it wins marketing approval for its sickle cell disease candidate therapy sevuparin.
Sevuparin, currently in Phase II trials in "core" sickle cell countries bordering Africa, is a modified low-molecular weight heparin which has no anti-coagulant effect anymore but is able to prevent adhesion of red and white blood cells to the vessel wall. By this mechanism blood flow is preserved and the sickling process can be prevented
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