Pharmaceutical Technology on MSN15d
FDA approves Mirum’s Ctexli for cerebrotendinous xanthomatosis
Mirum Pharmaceuticals’ Ctexli (chenodiol) has gained US Food and Drug Administration (FDA) approval for treating adults with cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease. It is ...
US FDA approves Mirum Pharma's genetic disorder drug
The U.S. Food and Drug Administration approved Mirum Pharmaceuticals' genetic disorder drug on Friday, making it the first treatment for a rare condition that interferes with the body's ability to ...
BioSpace15d
Mirum Establishes Liver Expertise With First FDA Nod for ‘Very Rare’ Disease
Ctexli’s approval further entrenches Mirum as a leader in rare liver diseases, alongside its cornerstone product Livmarli and upcoming drug volixibat.
Deal pays off for drugmaker with approval for rare cholesterol-related disease treatment
A young Peninsula drugmaker won regulatory approval Friday for a rare-disease drug picked up in a deal two years ago.
The Pharma Letter15d
FDA approves first treatment for cerebrotendinous xanthomatosis
The US Food and Drug Administration (FDA) late Friday revealed it has approved Ctexli (chenodiol) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Developed by Mirum ...
Yahoo Finance18d
FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease
SILVER SPRING, Md., Feb. 21, 2025 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Ctexli (chenodiol) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults.
Devdiscourse16d
Health Alerts: Impact of US Aid Freeze and Breakthroughs in Modern Medicine
Recent health news highlights disruptions in HIV and malaria supply chains due to a U.S. aid freeze, FDA's approval of a new ...
Endocrinology Advisor14d
FDA Approves Ctexli for Cerebrotendinous Xanthomatosis
The Food and Drug Administration has approved Ctexli (chenodiol) for the treatment of cerebrotendinous xanthomatosis in adults.
Business Insider18d
FDA approves Mirum treatment for cerebrotendinous xanthomatosis
The Food and Drug Administration approved Ctexli for the treatment of cerebrotendinous xanthomatosis in adults. “Ctexli is the first FDA-approved drug to treat CTX, a very rare lipid storage ...
Healio19d
FDA approves first treatment for rare lipid storage disease
Mirum Pharmaceuticals received first treatment approval for treatment of cerebrotendinous xanthomatosis. Ctexli resulted in significant reduction in plasma cholestanol and urine 23S-pentol.
Yahoo Finance15d
FDA approves Mirum’s Ctexli for cerebrotendinous xanthomatosis
Mirum Pharmaceuticals’ Ctexli (chenodiol) has gained US Food and Drug Administration (FDA) approval for treating adults with cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease.
Monthly Prescribing Reference19d
FDA Approves Ctexli for Cerebrotendinous Xanthomatosis
Ctexli 250mg 3 times daily showed significant reductions in plasma cholestanol and urine 23S-pentol compared with placebo. The Food and Drug Administration (FDA) has approved Ctexli ™ (chenodiol ...