Odevixibat

Unassigned

New Medicines

Progressive familial intrahepatic cholestasis (PFIC)

Information

New molecular entity
Albireo
Albireo

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Oct 18: Odevixibat receives Fast Track designation in US for PFIC [5] Mar 17: According to Albireo pipeline, PII studies have started [2].


Mar 17: Has orphan drug status in the US & EU [2]. 10/03/2017 12:17:57


Nov 16: Granted PRIME (PRIority MEdicines) status in the EU for treatment of PFIC. Albireo plans to initiate a pivotal clinical trial in PFIC in the US and Europe, and anticipates meeting with the US FDA regarding the planned trial in Q1 17 [2].

Category

An inhibitor of the ileal bile acid transporter (IBAT; also called the apical sodium-dependent bile acid transporter).
Estimated prevalence of PFIC varies between 1 in 50,000 and 1 in 100,000 [1].
Progressive familial intrahepatic cholestasis (PFIC)
Oral

Trial or other data

Sep 18: Phase III PEDFIC 2 trial (NCT03659916) initiated in the EU and US to evaluate long term safety and efficacy of odevixibat in children with PFIC types 1 and 2. [4]


May 18: First patient enrolled in the Pivotal phase III PEDFIC-1 trial (NCT03566238) of odevixibat in patients with PFIC. The randomised, double-blind, placebo-controlled trial will enrol approximately 60 patients in the EU and US [3]


Feb 15: Albireo announces results from a PI trial that evaluated the safety, pharmacokinetics and pharmacodynamics of A4250 in healthy volunteers [2].

Evidence based evaluations

NIHR