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Which regulatory body will grant the next major approval for Miplyffa outside of the United States by September 30, 2025?
2
Resolution / Starting Odds
European Medicines Agency (EMA) • 25%
Health Canada • 25%
Japan's PMDA • 25%
Other • 25%
Official announcements from regulatory bodies or Zevra Therapeutics
Story
FDA Approves Zevra's Miplyffa for Niemann-Pick Disease Type C, $150 Million Voucher
Sep 20, 2024, 01:40 PM
The U.S. Food and Drug Administration (FDA) has approved Zevra Therapeutics' drug, arimoclomol, also known as Miplyffa, for the treatment of Niemann-Pick disease type C (NPC), a rare and fatal genetic disorder. This approval marks the first oral medication to receive FDA approval for NPC. In addition, Zevra Therapeutics is now eligible to receive a Priority Review Voucher worth approximately $150 million. The approval was announced on September 20, 2024, ahead of the NDA PDUFA date of September 21, 2024.
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FDA only • 25%
EMA only • 25%
Both FDA and EMA • 25%
Neither • 25%
Yes • 50%
No • 50%
FDA (USA) • 25%
EMA (Europe) • 25%
MHRA (UK) • 25%
Other • 25%
1-3 countries • 25%
4-6 countries • 25%
7-9 countries • 25%
10 or more countries • 25%
European Medicines Agency (EMA) • 25%
Medicines and Healthcare products Regulatory Agency (MHRA) • 25%
Therapeutic Goods Administration (TGA) • 25%
Other • 25%
Only in the US • 25%
Only in the EU • 25%
In both US and EU • 25%
In US, EU, and Japan • 25%
FDA (USA) • 25%
EMA (Europe) • 25%
MHRA (UK) • 25%
Other • 25%
Only FDA • 25%
FDA and EMA • 25%
FDA, EMA, and MHRA • 25%
None • 25%
European Union • 25%
Japan • 25%
Canada • 25%
Other • 25%
European Union • 25%
United Kingdom • 25%
Australia • 25%
Other • 25%
Yes • 50%
No • 50%
FDA (USA) • 25%
EMA (EU) • 25%
MHRA (UK) • 25%
Other • 25%