PHEBURANE (sodium phenylbutyrate) - Urea cycle disorders

Opinions on drugs - Posted on Jan 09 2025

Reason for request

Initial inclusion

Summary of opinion

Favourable opinion for reimbursement as adjunctive therapy in the chronic management of urea cycle disorders, involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase or argininosuccinate synthetase (see the complete wording of the MA indication).

No clinical added value of the new oral solution form compared to the granule form of PHEBURANE (sodium phenylbutyrate) already available.

Clinical Benefit

Substantial

The clinical benefit of PHEBURANE 350 mg/ml (sodium phenylbutyrate) oral solution is substantial in the MA indication.

Clinical Added Value

no clinical added value

PHEBURANE 350 mg/ml (sodium phenylbutyrate) oral solution is a range supplement that does not provide any clinical added value (CAV V) compared to the form already listed.