An experimental treatment for Alagille syndrome (ALGS) has been approved by the US Food and Drug Administration (FDA) for another rare liver disease, called progressive familial intrahepatic cholestasis (PFIC). The approval marks the first approved treatment for PFIC and could help pave the way for a future ALGS indication.

The drug, odevixibat (Bylvay™), is a potent, nonsystemic ileal bile acid inhibitor approved to treat pruritus in PFIC patients. Pruritus is also a common symptom of ALGS and odevixibat is currently being investigated in a phase 3 clinical trial, called ASSERT (NCT04674761), for the treatment of pruritus in ALGS patients. Topline results of the ASSERT trial are expected in 2022.

The approval of odevixibat for PFIC was supported by data from two clinical trials, PEDFIC1 (NCT03566238) and PEDFIC2 (NCT03659916). Both studies showed that odevixibat successfully reduced pruritus and serum bile acid levels while being well-tolerated with a low incidence of diarrhea or bowel movement side effects. In the PEDFIC1 study, odevixibat was able to significantly improve pruritus compared to placebo (P =.004) while also significantly reducing serum bile acid levels (P =.003). In addition to effects on pruritus and bile acid levels, odevixibat also improved growth and other markers of liver function in patients in the PEDFIC2 trial over a 48-week period. No serious treatment-related adverse events were reported in the studies.

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Odevixibat is approved as a once-daily oral capsule but can be opened and mixed with food, if needed. The recommended dose of odevixibat is 40 mcg/kg which can be increased up to 120 mcg/kg if there is insufficient clinical response after 3 months. The European Commission has also granted marketing authorization for odevixibat. 

The success of these trials may translate to ALGS patients. Ron Cooper, president and CEO of Albireo Pharma, the manufacturer of odevixibat, said, “We’re humbled by the children, families and investigators whose commitment to our clinical trials will bring hope and treatment benefit for so many future patients.”


MULTIMEDIA UPDATE – Albireo announces FDA approval of BylvayTM (odevixibat), the first drug treatment for patients with progressive familial intrahepatic cholestasis (PFIC). News release. Albireo Pharma; July 20, 2021.

Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT). US National Laboratories of Medicine. Accessed July 21, 2021.