10:34 AM EDT, 06/28/2024 (MT Newswires) -- Mirum Pharmaceuticals ( MIRM ) said Friday that it has submitted a new drug application for chenodiol for treating patients with cerebrotendinous xanthomatosis, or CTX, in the US.
CTX is a rare progressive disorder of cholesterol metabolism that can affect the brain, spinal cord, tendons, eyes, and arteries, the company said.
The submission of the application was backed by the results from a phase 3 trial of chenodiol in adults with CTX. The study met its primary endpoint of reduction in bile alcohols and showed that treatment with chenodiol significantly improved serum cholestanol, the company added.
Shares of Mirum Pharmaceuticals ( MIRM ) rose 2% in recent trading.
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