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FDA Alerts
Genetic and Congenital

Mirum Pharmaceuticals submits new drug application for Chenodiol for CTX

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Mirum Pharmaceuticals announced the submission of a New Drug Application to the U.S. Food and Drug Administration for chenodiol for the treatment of cerebrotendinous xanthomatosis (CTX), according to a press release. CTX is a rare genetic disorder affecting cholesterol metabolism, leading to severe symptoms and neurologic deterioration.

The submission follows positive results from the Phase 3 RESTORE study, which showed significant reduction in bile alcohols and improved serum cholestanol levels.

If approved, chenodiol would be the first FDA-approved treatment for CTX in the U.S.

“When you have CTX, timely diagnosis and treatment can have a transformative impact on the lives of people living with this disease,” said Jean Pickford, executive director, CTX Alliance. “We were excited about the RESTORE data and are hopeful that the chenodiol submission to the FDA will result in an approval, enabling faster access to treatment and helping patients and their families earlier in their disease journey.”

Read the full press release here. 

 

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