Phase 3 Trial of Experimental Progressive Familial Intrahepatic Cholestasis Drug for Pediatric Patients Begins

According to a story from, the biopharmaceutical company Mirum Pharmaceuticals recently announced that they have begun dosing the first patient in a phase 3 clinical trial. This clinical trial will test Mirum’s lead investigational product candidate as a treatment for progressive familial intrahepatic cholestasis (PFIC) in child patients. As there are currently no approved therapies for this rare disease, positive results in this study could be groundbreaking for patients with this illness.

About Progressive Familial Intrahepatic Cholestasis (PFIC)

Progressive familial intrahepatic cholestasis (PFIC) are a group of hereditary diseases in which the flow of bile is affected. There are several different type of PFIC which are linked to different genetic mutations. These mutations generally cause a defect affecting biliary epithelial transporters. The symptoms of these diseases begin to appear in childhood, but some patients are not diagnosed until years later. These symptoms include lack of normal bile flow, cirrhosis, failure to thrive, jaundice, fat malabsorption, and severe itching. PFIC can eventually lead to complications such as osteopenia, a condition of lowered mineral density in bones. Treatment is generally supportive and symptomatic. Medications used to relieve symptoms include ursodeoxycholic acid, naloxone, and rifampin. Other approaches include certain surgical procedures and vitamin supplementation. If liver function begins to decline, a transplant may be necessary. To learn more about PFIC, click here.

About Maralixabat

Maralixabat is an experimental drug that is designed to prevent the overload of bile acid, which is a major contributor to symptoms experienced by patients with PFIC, including debilitating itching (known as pruritus) and liver damage. The drug is also being developed for other liver diseases with similar mechanisms, such as Alagille syndrome. Results in an earlier phase 2 study were encouraging, and maralixabat was able improve severe itching and speed up the pace of growth in pediatric patients.

The trial will compare the impact of maralixabat alongside a placebo control for a period of six months. This international trial, which will include study centers in the US, Europe, Canada, Asia, the Middle East, and Latin America, will include a total of 30 pediatric patients that will range between the ages of one and seventeen years. There is also provision for a supplementary cohort of patients that did not meet the initial inclusion criteria.

Hopefully, the results of this trial will bode well for the PFIC patient community.

Share this post

Share on facebook
Share on google
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email