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Alagille Syndrome
Journal Scan

FDA approves first drug for patients with progressive familial intrahepatic cholestasis

Posted on July 21, 2021
FDA approves new therapy for rare disease affecting optic nerve, spinal cord

The U.S. Food and Drug Administration has approved odevixibat (Bylvay; Albireo Pharma, Inc) for the treatment of pruritus in all subtypes of progressive familial intrahepatic cholestasis (PFIC), according to a press release. Odevixibat is a non-systemic ileal bile acid transport inhibitor (IBATi).

Approval was based on data from two phase 3 trials which demonstrated treatment with odevixibat resulted in sustained reduction in serum bile acids in addition to improvements in pruritus assessments, growth, and other markers of liver function in patients treated up to 48 weeks.

“Treating children with PFIC can be difficult and frustrating given the current treatment options. Bylvay gives us a non-surgical option and will change how we treat PFIC,” said Richard Thompson, Professor of Molecular Hepatology at King’s College London and principal investigator of PEDFIC 1 and PEDFIC 2 in the press release. “With this approval, my colleagues and I now have the opportunity to revisit how PFIC patients are being managed and we are hopeful for better outcomes for these children.”

Odevixibat is currently being studied in the ASSERT Phase 3 clinical trial in patients with Alagille syndrome. Topline data from that trial is expect in 2022.

Read the full press release here.

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