Reason for request
Inclusion
Clinical Benefit
| Substantial |
The actual benefit of PHEBURANE is considerable.
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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.
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