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Alagille Syndrome
FDA Alerts

FDA approves maralixibat for treatment of cholestatic pruritus in ALGS

Posted on September 29, 2021
FDA Grants DSG3-CAART Orphan Drug Designation for the Treatment of Pemphigus Vulgaris

The U.S. Food and Drug Administration has approved maralixibat (Livmarli; Mirum Pharmaceuticals) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) who are ≥ 1 year of age. This is the first FDA-approved medication for patients with ALGS.

Maralixibat is a minimally absorbed ileal bile acid transporter inhibitor.

“Children with Alagille syndrome suffer from cholestatic pruritus, which is serious, unremitting, and debilitating. Their sleep is disrupted, and they endure bleeding and scarring of the skin due to unrelenting scratching,” said Binita M. Kamath, MBBChir, Pediatric Hepatologist, The Hospital for Sick Children (SickKids), Toronto, Ontario, Canada in a press release. “There have been no approved treatments to date for cholestatic pruritus in Alagille syndrome, and many children ultimately require major surgical interventions such as liver transplantation for refractory pruritus. The approval of LIVMARLI signifies a meaningful shift in the treatment paradigm for Alagille syndrome and provides hope for the many families who have lived with persistent itch for far too long.”

Read the full press release here.

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