Accepted for priority review with Prescription Drug User Fee Act (PDUFA) action date of August 17, 2026--If approved, ...
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FDA accepts Beren Therapeutics’ NDA for Niemann-Pick disease
The submission includes data indicating a meaningful survival benefit for infantile-onset NPC patients treated with adrabetadex.
Dublin, Aug. 01, 2025 (GLOBE NEWSWIRE) -- The "Niemann-Pick Disease Type C NPC Market - A Global and Regional Analysis: Focus on Country and Region - Analysis and Forecast, 2025-2035" report has been ...
Niemann-Pick Disease Type C (NPC), an ultra-rare, progressive neurodegenerative disease, is heterogeneous and is often misdiagnosed or undiagnosed. A recent study aimed to get a fuller picture of the ...
A Zevra Therapeutics drug is now the first FDA-approved therapy for an ultra-rare, inherited metabolic disorder whose effects on the central nervous system can become fatal by the time a patient is a ...
CELEBRATION, Fla., Dec. 29, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with ...
LEIDEN, Netherlands--(BUSINESS WIRE)--Azafaros has announced that data from the ongoing double-blind, placebo-controlled Phase 2 RAINBOW study investigating its lead asset, nizubaglustat in patients ...
After gaining FDA approval for Miplyffa ® (arimoclomol) as the first-ever therapy indicated to treat Niemann-Pick disease type C (NPC), Zevra Therapeutics has cashed in on the regulatory win by ...
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