Vanda Pharmaceuticals (VNDA) Receives Orphan Drug Designation for Imsidolimab

VNDA Stock Gains After-Hours As FDA Grants Rare Pediatric Disease Designation For Experimental Therapy

The designation covers VCA-894A, Vanda’s investigational treatment for Charcot-Marie-Tooth disease type 2S.

FDA Accepts Immunome’s NDA for Varegacestat in Desmoid Tumors, Setting Up an April 2027 Decision

Key Takeaways The FDA has accepted Immunome’s New Drug Application (NDA) for varegacestat in adults with desmoid tumors, with a PDUFA target

TolerogenixX Has Received EMA Orphan Drug Designation for MIC-Lx, Strengthening its Regulatory Pathway in Kidney Transplantation

Supporting the development of its proprietary MIC-Lx Modified Immune Cells technology as a novel approach to reduce lifelong systemic immuno

Study finds significant discordance in orphan drug approvals between U.S. FDA and European Medicines Agency (2011-2023)

In a retrospective analysis, Jin Ding and colleagues explore the discrepancy in regulatory authorization of orphan drugs by U.S. and Europea

Sun Pharma gets go-ahead to sell generic semaglutide in South Africa

The approval is to treat Type II diabetes

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