GLI Commends Repurposed Livmarli’s FDA Approval for Patients over 5 with PFIC

(Washington, D.C. March 14, 2024) –Global Liver Institute (GLI) celebrates a win for rare disease patients with the breaking news of the U.S. FDA’s approval for Mirum Pharmaceuticals’ Livmarli to treat cholestasis pruritus in patients 5 and older that have been diagnosed with progressive familial intrahepatic cholestasis (PFIC), a rare, genetic disorder in which bile builds up in the liver.

“This repurposing of an already-approved medication brings hope to a population of patients who have had limited options for treatment, like so many others with a rare disease,” shared Donna R. Cryer, JD, founder and CEO of GLI. “Instead of facing certain disease progression to end-stage liver disease and the necessity of transplant, a renewed quality of life and longevity is now available for patients with PFIC.”

The oral solution was previously approved in 2021 to treat cholestatic pruritus in patients ages 3 months and older with Alagille Syndrome (ALGS) and is now available to patients with two of the three rare pediatric liver diseases that cause pruritus. Pruritus is the intense itch that accompanies some rare, genetic liver disorders, causing a myriad of physical and psychological effects on patients that impact their health and daily living. 

• •  PFIC is estimated to affect 1 in 50000-100000 births. 
  •  PFIC and ALGS cause a buildup of bile acid in the liver, eventually requiring patients to undergo transplants to stay alive.
  •  Life expectancy is shorter with PFIC, with only 50% of patients surviving past their 10th birthday.

GLI’s Program Director of Pediatric and Rare Liver Diseases, Kristin Hatcher, noted  “With only 5% of rare diseases in the United States today with an approved drug for their disease, this approval brings hope to many other rare disease patients to know that molecules are being developed, or repurposed, for rare diseases in general. We have not been forgotten.”

About Global Liver Institute  

Global Liver Institute (GLI) is a 501(c)3 nonprofit organization founded in the belief that liver health must take its place on the global public health agenda commensurate with the prevalence and impact of liver illness. GLI promotes innovation, encourages collaboration, and supports the scaling of optimal approaches to help eradicate liver diseases. Operating globally, GLI is committed to solving the problems that matter to liver patients and equipping advocates to improve the lives of individuals and families impacted by liver disease. GLI holds Platinum Transparency with Candid/GuideStar, is a member of the National Health Council, and serves as a Healthy People 2030 Champion. Follow GLI on Facebook, Instagram, LinkedIn, and YouTube or visit www.globalliver.org.