The United States Food and Drug Administration has granted Fast Track designation to a drug being researched as a potential treatment for progressive familial intrahepatic cholestasis, a serious liver disease with limited treatment options. For more information about this news, you can click here to see the source press release by Albiero Pharma.
About Progressive Familial Intrahepatic Cholestasis (PFIC)
PFIC is a form of liver disease that mainly affects children. The disorder progresses over time and may become life-threatening. PFIC affects the ability of liver cells to secrete bile, a fluid that aids digestion. As a result, bile builds up in liver cells, which causes damage.
A Potential Drug
Albireo is developing an investigational drug that may become a new treatment for PFIC. The drug, called A4250, is believed to work by selectively inhibiting the ileal bile acid transporter locally in the gut. There are also plans to think about developing A4250 for other pediatric cholestasis liver diseases in the future.
The drug is currently being studied in a Phase 3 clinical trial of patients with PFIC types 1 and 2. According to Albireo’s website, the trial compares the effects of A4250 to a placebo in a double-blind, randomised study. It’s taking place across multiple centres in the US, Canada, Europe, the Middle East, and Australia, and is designed to enrol about 60 patients with PFIC types 1 or 2 who are aged between 6 months and eighteen years. The program also includes an open-label extension study.
A4250 has previously been granted several designations to support its development for patients with PFIC. The FDA has awarded the Orphan Drug and Rare Pediatric Disease statuses (with the possibility of applying for a Priority Review Voucher), and the European Medicines Agency has given it Orphan Drug Designation, access to the PRIME program, and agreed to A4250’s Paediatric Investigation Plan.
In addition to these, the FDA has now also awarded the drug Fast Track designation. This designation is used to support the development, and speed up the review process, for drugs that would fulfil an unmet need for patients with serious conditions. Click here to find out more about Fast Track status.
In addition to PFIC, A4250 is also being studied as a potential treatment for other rare pediatric liver diseases, and the FDA has recently awarded Orphan Drug designation for its use in patients with Alagille syndrome, which you can read more about here.