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FDA Panel Backs Zevra's Arimoclomol For Rare Genetic Disorder, Analyst Highlights Balanced Portfolio Of Marketed Products And Pipeline Assets Focused On Rare Diseases
Friday, the FDA’s Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted in favor of Zevra Therapeutics, Inc.’s (NASDAQ:ZVRA) arimoclomol. The committee voted favorably (11 yes ...
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FDA Convenes Expert Panel Meeting For Zevra Therapeutics' Arimoclomol, Analyst Optimistic About Approval Despite Small Patient Base
The application for arimoclomol has been assigned a Prescription Drug User Fee Act (PDUFA) action date of September 21, which was extended from June 21. Previously known as KemPharm, Zevra ...
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FDA panel backs Zevra's arimoclomol, after earlier rejection
An FDA advisory committee has recommended approval of Zevra Therapeutics' arimoclomol for the ultra-rare disorder Niemann-Pick disease, three years after the regulator rejected the drug.
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Orphazyme 'looking at options' after lead drug hits buffers in EU
Arimoclomol was also being tested in another disease – inclusion body myositis (IBM) – but failed to move the needle in that indication in a phase 2/3 trial reported last year. It is Orphazyme ...
Business Insider8d
XOMA Royalty Reports Third Quarter 2024 Financial Results and Highlights Recent Activities
Zevra’s MIPLYFFA™ (arimoclomol) received FDA approval and became the sixth commercial asset in XOMA Royalty’s portfolio XOMA Royalty acquired a 50 percent economic interest in TWIST Bioscience’s ...