The U.S. Food and Drug Administration (FDA) has accepted Albireo Pharmaceuticals’ New Drug Application (NDA) for odevixibat for the treatment of pruritus in patients with progressive familial intrahepatic cholestasis (PFIC).
The FDA is scheduled to make a decision on the drug by July 20, 2021.
PFIC is a rare genetic disorder that causes progressive, life-threatening liver disease due to an inability to properly bile acids from the liver. The most common manifestation of PFIC is pruritus, which often results in a severely diminished quality of life. In many cases, this disease leads to cirrhosis and liver failure within the first 10 years of life, and nearly all PFIC patients require treatment before the age of 40 years. Currently, there are no drug therapies approved for PFIC. If approved, odevixibat will provide a once-daily, oral drug option for children with PFIC patients.
The NDA approval is supported by results from the PEDFIC 1 and PEDFIC 2 studies. PEDFIC 1 was the first and largest phase study conducted in PFIC, which evaluated the efficacy and tolerability of odevixibat in reducing pruritus and serum bile acids. In the PEDFIC 1 study, odevixibat met both the pruritus (p=0.004) and serum bile acid (p=0.003) primary endpoints and was well tolerated. PEDFIC 2 is a long-term, open-label phase 3 extension study of PEDFIC 1.
For more information about PFIC and other rare metabolic disorders, visit checkrare.com/diseases/metabolic-disorders/