California-based Mirum Pharmaceuticals Inc. has reportedly announced that its drug, Livmarli (Maralixibat) has become the first U.S. FDA approved therapy for treating itching (cholestatic pruritus) in patients suffering from Alagille Syndrome (ALGS), a rare liver disease.

For the unversed, ALGS is a genetic disease wherein bile ducts are abnormally malformed,  narrow, and low in number, leading to bile accumulation in the liver.

The drug developed by Mirum Pharma blocks an important bile acid transport (iBAT) protein on the surface of the small intestine which helps in removal of bile acid from the human body through feces. Besides the functioning, the drug’s approval comprises information regarding side effects, including changes in liver tests which are often an indication of liver injury.

The drug would supposedly be made available for distribution immediately under the brand name Livmarli and will be priced at approximately USD 1,550 per unit. The annual cost of drug administration for a child weighing over 17 kg (37.4 pounds) is estimated to be around USD 391,000.

According to sources, the closest competitor for Livmarli is Albireo Pharma's Bylvay, which is currently in its late-stage testing. In July, Bylvay received the U.S. FDA’s approval for treatment of another liver disease called Progressive Familial Intrahepatic Cholestasis (PFIC), with pricing of USD 385,000 a year.

Chief Executive Officer of Mirum Pharmaceuticals, Chris Peetz, was quoted saying that itching is really devasting for children and families dealing with Alagille Syndrome and is the driving factor for potential liver transplant decisions.

Experts reportedly said that Livmarli could amass USD 400 million in sales by 2030, while analysts from Mirum expect a substantially higher market opportunity of USD 500 million for the indication.

The dosage for Livmarli will be determined by physicians as per the patient’s weight, cited sources with reliable information.

Source credit: https://www.usnews.com/news/us/articles/2021-09-29/mirums-oral-drug-becomes-first-us-approved-alagille-syndrome-therapy