PROCYSBI (cysteamine), metabolism product

RARE DISEASE - New medicinal product

Opinions on drugs - Posted on Jan 08 2016

Reason for request

Inscription

Minor improvement in the treatment of proven nephropathic cystinosis by comparison with CYSTAGON.

PROCYSBI 25 mg and 75 mg have Marketing Authorisation in the treatment of proven nephropathic cystinosis.
The presentation takes the form of prolonged-release gastro-resistant hard capsules, which need to be taken only once every 12 hours.
It is an alternative to CYSTAGON (same active ingredient, but immediate release), which is the only other medicinal product with Marketing Authorisation in nephropathic cystinosis.
PROCYSBI was found to be non-inferior to CYSTAGON in terms of maintenance of intraleukocytic cystine levels in patients managed with CYSTAGON.

Clinical Benefit

Substantial

Le service médical rendu par PROCYSBI est important dans le traitement de la cystinose néphropathique confirmée.

Clinical Added Value

minor

La Commission considère que PROCYSBI, spécialité à base de bitartrate de cystéamine à libération prolongée, apporte une amélioration du service médical rendu mineure (ASMR IV) par rapport à CYSTAGON, formulation à libération immédiate du même principe actif.