Albireo Announces U.S. FDA Acceptance of New Drug Application for Odevixibat
January 25, 2021 at 08:00 AM EST
- Company receives FDA Priority Review with PDUFA date set for July 20, 2021 -
- Submission data for PFIC types 1, 2, 3, supporting once-daily use across a wide range of patients -
- FDA has granted odevixibat Fast Track, Rare Pediatric Disease and Orphan Drug Designations -
- Odevixibat on track to be first approved drug for PFIC patients -
BOSTON, Jan. 25, 2021 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage rare liver disease company developing novel bile acid modulators, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) for odevixibat for the treatment of pruritus in patients with progressive familial intrahepatic cholestasis (PFIC). Odevixibat is a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) being developed to treat patients with rare pediatric cholestatic liver diseases, including PFIC, biliary atresia and Alagille syndrome. The FDA has granted Priority Review and set a Prescription Drug User Fee Act (PDUFA) goal date of July 20, 2021, supporting the Company’s previous guidance of a planned launch in the second half of 2021. Odevixibat previously received Fast Track, Rare Pediatric Disease and Orphan Drug Designations in the U.S.
“With regulatory submissions completed in record time and now accepted for review by both the FDA and EMA, odevixibat has the potential to become the first approved drug treatment for patients with PFIC,” said Ron Cooper, President and Chief Executive Officer of Albireo. “Additionally, with no approved treatments, PFIC presents an exciting commercial opportunity and will pave the way for expected additional indications in Alagille syndrome and biliary atresia.”
PFIC is a rare and devastating disorder that causes progressive, life-threatening liver disease. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life. There are no drug therapies currently approved for PFIC, only surgical options. If approved, odevixibat will provide a once-daily, oral drug option for children with PFIC.
“As parents of children with PFIC, our focus has long been on consoling our children who scratch so hard they draw blood and on conversations about liver transplants,” said Emily Ventura, leader of PFIC Advocacy and Resource Network (www.pfic.org) and mother to a PFIC patient. “With this exciting decision from the FDA, we are one step closer to alleviating our children’s burden and bringing new hope to what was previously an uncertain future with no drug treatments available.”
The NDA submission is supported by results from PEDFIC 1 and PEDFIC 2 Phase 3 studies. PEDFIC 1 was the first and largest, global, pivotal Phase 3 study conducted in PFIC, which evaluated the efficacy and tolerability of odevixibat in reducing pruritus and serum bile acids in a randomized, double-blind, placebo-controlled trial. 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 with very low incidence of diarrhea/frequent bowel movements (9.5% of odevixibat treated patients vs. 5.0% of placebo patients). PEDFIC 2 is a long-term, open-label Phase 3 extension study.
In Europe, the Company has submitted odevixibat for a Marketing Authorization Application (MAA) to the EMA seeking approval in PFIC. Odevixibat is the only IBATi granted accelerated assessment by the EMA. It has also been granted Orphan Designation, as well as access to the PRIority MEdicines (PRIME) scheme for the treatment of PFIC. The EMA’s Pediatric Committee has agreed to Albireo’s odevixibat Pediatric Investigation Plans for PFIC and biliary atresia. In addition to PFIC, odevixibat has Orphan Drug Designations for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis.
With U.S. and EU regulatory submissions for odevixibat in PFIC completed, the Company anticipates potential regulatory approvals, issuance of a rare pediatric disease priority review voucher and launch in the second half of 2021. Odevixibat is also currently being evaluated in the BOLD Phase 3 trial in patients with biliary atresia, ASSERT Phase 3 trial in Alagille syndrome and the ongoing PEDFIC 2 Phase 3 open-label trial in patients with PFIC. The Company provides an Expanded Access Program for eligible patients with PFIC in the U.S., Europe, Canada and Australia.
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