Shares of a clinical-stage rare liver disease corporation that creates new bile acid modulators, Albireo Pharma, Inc. (ALBO) surged by +6.49% to close the trading at $39.05.
The company last week reported that it’s New Drug Application (NDA) for odevixibat has been approved by the U.S. Food and Drug Administration (FDA) to treat pruritus in patients with Progressive Family Intrahepatic Cholestasis (PFIC). Odevixibat has been developed to treat patients with rare pediatric cholestatic liver disorders, including PFIC, biliary atresia, and Alagille syndrome, as a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi). The FDA has given a Priority Review and set a target date of July 20, 2021, for the Prescription Drug User Fee Act (PDUFA), upholding the Company’s previous guidance for a projected introduction in the second half of 2021. Quick Route, Rare Pediatric Disorder, and Orphan Drug Designations were previously earned by Odevixibat in the U.S.
Odevixibat has the ability to become the first approved prescription therapy for patients with PFIC, with regulatory applications completed in record time and now authorized for approval by both the FDA and EMA, said Ron Cooper, President and Chief Executive Officer of Albireo. In addition, PFIC poses an interesting market potential with no licensed drugs and will pave the way for anticipated additional indications for Alagille syndrome and biliary atresia.
PFIC is a rare and debilitating illness that causes life-threatening, progressive liver disease. In many cases, within the first 10 years of life, PFIC leads to cirrhosis and liver failure. There are no existing accepted drug treatments for PFIC, just surgical solutions. If accepted, odevixibat would provide children with PFIC with a once-daily, oral drug option.
Odevixibat has sent a Marketing Authorization Application (MAA) in Europe to the EMA requesting PFIC permission. Odevixibat is the only IBATi that has obtained an advanced review from the EMA. Orphan status and admission to the PRIority Drugs (PRIME) program for the care of PFIC has also been granted. The Odevixibat Pediatric Investigation Proposals for PFIC and Biliary Atresia have been accepted by the EMA Pediatric Commission. For the prevention of Alagille syndrome, biliary atresia, and primary biliary cholangitis, odevixibat has Orphan Drug Designations in addition to PFIC.
With the conclusion of U.S. and EU regulatory applications for odevixibat in PFIC, the Organization anticipates future regulatory approvals, issuance of a priority evaluation voucher for rare pediatric diseases, and launch in the second half of 2021. Odevixibat is also currently being tested in Phase 3 BOLD trial of patients with biliary atresia, the Phase 3 Alagille syndrome trial of ASSERT, and the pending Phase 3 open-label PEDFIC 2 trial of patients with PFIC. Albireo Pharma, Inc. (ALBO) has an Extended Access Service in the U.S., Europe, Canada, and Australia for qualifying patients with PFIC.