KOLBAM (cholic acid), bile acid

RARE DISEASE - NEw medicinal product

Opinions on drugs - Posted on Sep 27 2017

Inclusion

KOLBAM has Marketing Authorisation in the treatment of congenital abnormalities of primary bile acid synthesis due to deficiency in sterol 27-hydroxylase (manifested by cerebrotendineous xanthomatosis, CTX), in 2- (or α-) methylacyl-CoA racemase (AMACR) or in cholesterol 7α-hydroxylase (CYP7A1), from one month of age.
The available clinical data do not make it possible to ensure that KOLBAM meets the clinical objectives sought for a treatment of CTX, in particular in terms of efficacy on neurological symptoms.
The clinical data available in the other two enzyme deficiencies are limited to a single patient per deficiency. While the disease is rare, more patients could have been analysed.