Cholbam (cholic acid)
Bile Acid Synthesis Defects (e.g., peroxisomal disorders including Zellweger spectrum disorders)
Key Facts
About Asklepion Pharmaceuticals
Asklepion Pharmaceuticals is a mission-driven biotech dedicated to addressing the unmet medical needs of children with rare and critical diseases. Founded in 2006, the company leverages partnerships and a focused pipeline to advance therapies from concept to commercialization. Key achievements include an FDA-approved product (Cholbam), Orphan Drug Designation for its lead candidate, a Special Protocol Assessment (SPA) with the FDA for its Phase 3 program, and recognition from the National Organization for Rare Disorders (NORD). Its strategic direction centers on advancing IV citrulline through late-stage trials for a severe pediatric complication.
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