KAYFANDA (odevixibat) - Cholestatic pruritus in Alagille syndrome
Opinions on drugs -
Posted on
Jan 31 2025
Reason for request
First listing
Summary of opinion
Favourable opinion for reimbursement in “the treatment of cholestatic pruritus in Alagille syndrome (ALGS) in patients aged 6 months or older.”
Clinical Benefit
Substantial
The Committee deems that the clinical benefit of KAYFANDA (odevixibat) is substantial in the MA indication.
Clinical Added Value
moderate
Considering:
the results of a randomised, placebo-controlled, phase 3 trial conducted in children with a rare disease, demonstrating a clinically relevant effect in terms of reducing pruritus (reduction in scratching severity score) in 52 patients;
the safety profile of odevixibat;
but also the absence of data on cholestasis complications and recourse to transplantation due to refractory pruritus,
the Committee deems that KAYFANDA (odevixibat) provides a moderate clinical added value (CAV III) in the current care pathway.
Listen
