Acer wins FDA OK for Olpruva to treat urea cycle disorders

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The U.S. Food and Drug Administration (FDA) has greenlighted Olpruva, being developed by Acer Therapeutics (NASDAQ:ACER) and Swiss biotech Relief Therapeutics (OTCQB:RLFTF) (OTCQB:RLFTY) as a treatment for patients with urea cycle disorders (UCD).

The oral treatment, also known as sodium phenylbutyrate, will therefore be indicated in the U.S. as an adjunctive therapy to the standard of care for chronic management of adults and children with UCDs.

UCDs are a genetically-driven group of diseases that impact the urea cycle, leading to excess accumulation of blood ammonia, which can result in lethargy, coma, multi-organ failure, seizures, and psychiatric symptoms.

In June 2022, the FDA rejected the therapy citing the need for an adequate review of Acer's (ACER) third-party contract manufacturer for the drug, then known as ACER-001.

However, the following month, FDA accepted the company's resubmitted New Drug Application for ACER-001, issuing Jan. 15, 2023, as the target action date.

In August, Acer (ACER) and Relief (OTCQB:RLFTF) announced the European orphan medicinal product designation for ACER-001 as a treatment for the rare metabolic disorder Maple Syrup Urine Disease.