Mirum Submits New Drug Application To FDA For Chenodiol For Treatment Of CTX
Portfolio Pulse from Benzinga Newsdesk
Mirum Pharmaceuticals has submitted a New Drug Application (NDA) to the FDA for chenodiol, aimed at treating cerebrotendinous xanthomatosis (CTX). This submission is based on positive results from the Phase 3 RESTORE study. If approved, chenodiol would be the first and only therapy indicated for CTX in the US. Mirum holds orphan designation for this drug.

June 28, 2024 | 12:36 pm
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Mirum Pharmaceuticals has submitted an NDA to the FDA for chenodiol to treat CTX, based on positive Phase 3 results. If approved, it will be the first and only therapy for CTX in the US.
The submission of an NDA to the FDA is a significant milestone for Mirum Pharmaceuticals. The positive Phase 3 results and the potential to be the first and only therapy for CTX in the US are likely to boost investor confidence and positively impact the stock price in the short term.
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