NDA Filed for Urea Cycle Disorders Drug

Acer Therapeutics and Relief Therapeutics have submitted an NDA for ACER-001 (sodium phenylbutyrate) for treating urea cycle disorders (UCDs). The companies describe ACER-001 as a nitrogen-binding agent in development for use as adjunctive therapy in the chronic management of patients with UCDs involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase, or argininosuccinic acid synthetase.

The 505(b)(2) NDA submission is supported by data from two bioequivalence trials in which the therapy reportedly showed similar relative bioavailability for both phenylbutyrate and phenylacetate, the active metabolite of sodium phenylbutyrate, compared to Buphenyl (sodium phenylbutyrate).

The companies say UCDs are a group of disorders caused by genetic mutations that result in a deficiency in one of the six enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the bloodstream (hyperammonemia).