Friday, 19 April, 2024
HomeNews UpdateFDA approves expanded use of liver disease drug

FDA approves expanded use of liver disease drug

The US Food and Drug Administration (FDA) has approved the expanded use of Mirum Pharmaceuticals’ medication, Livmarli, to treat itching caused by a liver disorder in patients aged five and older.

The approval allows the drug to be used for treating itching caused by progressive familial intrahepatic cholestasis (PFIC), a genetic disorder that causes progressive liver disease typically leading to liver failure.

Reuters reports that Livmarli is already approved for treating itching caused by another type of rare genetic liver disorder known as Alagille Syndrome (ALGS).

“The drug will be launched with immediate availability for eligible patients in the US with the same pricing as it is for the approved ALGS indication,” the company said.

The FDA approval was based on data from a late-stage trial of 93 PFIC patients aged between 12 months and 17 years, where the drug helped reduce the severity of itching.

Mirum has also submitted an application to the FDA to expand the use of a higher concentration formulation of Livmarli in younger patients with PFIC.

The California-based drugmaker has been betting on its Livmarli franchise, seeking potential expansions into multiple liver disease indications.

 

Reuters article – US FDA approves expanded use of Mirum’s liver disease drug (Open access)

 

See more from MedicalBrief archives:

 

98% of rare diseases estimated to go undiagnosed in SA — advocacy group

 

FDA simplifies trials for rare paediatric disorders

 

 

 

 

 

MedicalBrief — our free weekly e-newsletter

We'd appreciate as much information as possible, however only an email address is required.