The Food and Drug Administration (FDA) has accepted for Priority Review the supplemental New Drug Application (sNDA) for odevixibat, an ileal bile acid transporter inhibitor, for the treatment of Alagille syndrome.
The sNDA is supported by data from the randomized, double-blind, placebo-controlled phase 3 ASSERT study (ClinicalTrials.gov Identifier: NCT04674761), which included 63 patients less than 17 years of age with a genetically confirmed diagnosis of Alagille syndrome. Patients were randomly assigned to receive either odevixibat or placebo orally once daily for 24 weeks.
Results showed that treatment with odevixibat met the primary endpoint demonstrating a statistically significant reduction in pruritus at month 6, as measured by the PRUCISION Observer-Reported Outcome scratching score, compared with placebo (P =.002). Additionally, a statistically significant reduction in serum bile acid concentration (secondary endpoint) from baseline to the average of weeks 20 and 24 was observed with odevixibat vs placebo (P =.001).
Odevixibat was well tolerated in the study; the incidence of overall adverse events was similar to placebo. Drug-related diarrhea was reported in 11.4% of odevixibat-treated patients and 5.9% of placebo-treated patients.
Odevixibat is currently marketed under the brand name Bylvay® for the treatment of progressive familial intrahepatic cholestasis. The application for the Alagille syndrome indication has been assigned a Prescription Drug User Fee Act target date of June 15, 2023.
- FDA grants June 15, 2023 PDUFA date to Albireo for Bylvay® in Alagille syndrome. News release. Albireo Pharma, Inc. Accessed February 14, 2023. https://www.globenewswire.com/news-release/2023/02/14/2607628/0/en/FDA-Grants-June-15-2023-PDUFA-Date-to-Albireo-for-Bylvay-in-Alagille-Syndrome.html.
- Bylvay® (odevixibat) data presented at AASLD The Liver Meeting® 2022, demonstrating native liver survival in children across PFIC types. News release. Albireo Pharma, Inc. November 7, 2022. Accessed February 14, 2023. https://ir.albireopharma.com/news-releases/news-release-details/bylvayr-odevixibat-data-presented-aasld-liver-meetingr-2022.