MIPLYFFA® and Zevra Therapeutics presented new clinical data on a drug targeting the underlying pathology of Niemann-Pick disease type C at the International Congress of Inborn Errors of Metabolism.
4 reports, 1 independent
Updated Sep 4
Gone quiet
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- 4
- Developments
- 4
- Repetition
- 50%
New informationRepeats or wire copies
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What happened
MIPLYFFA® and Zevra Therapeutics presented new clinical data on a drug targeting the underlying pathology of Niemann-Pick disease type C at the International Congress of Inborn Errors of Metabolism.
How it developed
Newest first. Tap a step to see who reported it.- Zevra Therapeutics, which treats NPC with MIPLYFFA, announced that the enrollment for the drug included patients in Puerto Rico. Neil McFarlane is the company's CEO.Sub-event
- The FDA approved Miplyffa for the treatment of Niemann-Pick disease.Sub-event
Company presented efficacy data on NPC drug at international congress.1 source
NPC, in partnership with Zevra Therapeutics, submitted a Marketing Authorisation Application to the European Medicines Agency for arimoclomol, a therapy for Niemann-Pick disease type C.1 source
Coverage
Newest first; wire copies grouped- GlobeNewswireZevra Therapeutics Presents Positive New Data Supporting Foundational Role of MIPLYFFA® (arimoclomol) for the Treatment of Niemann-Pick Disease Type C at the International Congress of Inborn Errors of
- GlobeNewswireZevra Reports Second Quarter 2025 Financial Results and Corporate Update Q2 2025 net revenue of $25.9 million, driven by product net revenue of $21.8 million Completed sale of PRV for $150.0 million,
- GlobeNewswireZevra Therapeutics Announces MIPLYFFA® and OLPRUVA® Presentations at the Annual Meeting of the Southeastern Regional Genetics Group (SERGG) CELEBRATION, Fla., July 18, 2025 (GLOBE NEWSWIRE) -- Zevra
- GlobeNewswireZevra Therapeutics Announces Open Label Extension Data Showing Sustained Long-Term Efficacy of MIPLYFFA for the Treatment of NPC Published in the Journal of Molecular Genetics and Metabolism CELEBRAT