According to a press release from the rare liver disease company Albireo Pharma, Inc., the US Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for odevixibat. This experimental therapy is being developed as a treatment for pruritus (itching) associated with progressive familial intrahepatic cholestasis (PFIC), a rare disease. Under Priority Review, the FDA will set to make a decision on this drug by July 20, 2021.
About Progressive Familial Intrahepatic Cholestasis
Progressive familial intrahepatic cholestasis (PFIC) are a group of hereditary diseases in which the flow of bile is affected. There are several different types 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.
Odevixibat is on track to being the first ever treatment for this disease to receive approval from the FDA. It is classified as a ileal bile acid transport inhibitor, designed to be taken once per day. The NDA for this therapy was filed following promising results in phase 3 trials. The drug was able to meet both primary endpoints of reduction in itching and in serum bile acid. This investigational treatment is also being trialed for other rare liver diseases, such as biliary atresia and Alagille syndrome.
This drug is also being evaluated for approval in the EU where it has earned Orphan Drug designation and PRIME designation. In the US, odevixibat has earned Orphan Drug designation, Rare Pediatric Disease designation, and Fast Track designation. If approved, this experimental treatment has the potential to offer progressive familial intrahepatic cholestasis patients of all ages an orally active, once daily therapy that can improve quality of life.