Reason for request

Inclusion

-


Clinical Benefit

Substantial

The actual benefit of PHEBURANE is considerable.


Clinical Added Value

minor

In view of :

- the pharmaceutical formulation, which is able to mask the unpleasant taste of sodium phenylbutyrate
- the clinical follow-up data of 17 patients treated with PHEBURANE under the terms of a temporary usage authorisation (TUA), who showed fewer episodes of vomiting and hyperammonaemic decompensation on PHEBURANE by comparison with AMMONAPS,

PHEBURANE offers a minor improvement in actual benefit (IAB IV) over AMMONAPS as adjunctive therapy in the long-term management of urea cycle disorders involving deficiency in carbamoyl phosphate synthetase, ornithine transcarbamylase or argininosuccinate synthetase.