FDA approves Olpruva for patients with urea cycle disorders

28 December 2022

The US Food and Drug Administration (FDA) has approved Olpruva (sodium phenylbutyrate; ACER-001) for oral suspension for the treatment of certain patients living with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).

Olpruva has been developed by USA-based Acer Therapeutics (Nasdaq: ACER) and its collaboration partner, Swiss firm Relief Therapeutics (RLF: SIX). Following the announcement yesterday, Relief’s shares rose 6.9% to 0.03 Swiss francs, while Acer stock slumped 22% to $2.90. The FDA approval comes ahead of the target date for a decision of January 15, 2023, so this should be welcome news for the companies.

In June this year, citing an incomplete contract manufacturer inspection, the FDA issued a complete response letter (CRL) rejecting Acer’s application for sodium phenylbutyrate for oral suspension in UCD. Acer resubmitted its New Drug Application (NDA) for the drug in July.

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