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Azafaros Secures € 132M in Financing to advance Phase 3 clinical programs, including NPC

13 May 2025 Leiden, Netherlands, 13 May 2025 – Azafaros, a clinical-stage company focused on developing disease-modifying therapeutics to offer new treatment options to patients with rare lysosomal storage disorders, announces the completion of an oversubscribed €132M Series B financing led by Jeito Capital, co-led by Forbion Growth and with additional participation from Seroba, Pictet Group …

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Zevra Therapeutics to Sell Priority-Review Voucher for $150 Million related to NPC drug approval

February 27, 2025 at 7:31 AM EST CELEBRATION, Fla., Feb. 27, 2025 (GLOBE NEWSWIRE) — Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that it has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease …

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FDA Approves 2nd New Drug to Treat NPC

FDA Approves New Drug to Treat Niemann-Pick Disease, Type C | FDA For Immediate Release:September 24, 2024 Today, the U.S. Food and Drug Administration approved Aqneursa (levacetylleucine) for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kilograms.  “This is the second treatment …