FDA Approves Acer and Relief’ Olpruva for Patients with Urea Cycle Disorders
December 27, 2022
The U.S. Food and Drug Administration approve Acer Therapeutics and Relief Therapeutics Olpruva for oral suspension for the treatment of certain patients with urea cycle disorders.
The approval covers deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OT), or argininosuccinic acid synthetase (AS).
Olpruva’s approval triggers the availability of a $42.5 million term loan to Acer under the previously announced March 2022 loan agreement the company entered into with affiliates of Marathon Asset Management. If Acer requests and receives the loan proceeds, management believes it will have sufficient resources to fund current operations into second half of 2023.
Urea cycle disorders are a group of rare, genetic disorders that can cause harmful ammonia to build up in the blood, potentially resulting in brain damage and neurocognitive impairments, if ammonia levels are not controlled. Any increase in ammonia over time is serious. People with the condition must adhere to dietary protein restrictions and have alternative medication options to help control ammonia levels.
Olpruva is used along with certain therapy, including changes in diet, for the long-term management of certain urea cycle disorders. It is not used to treat rapid increase of ammonia in the blood (acute hyperammonemia), which can be life-threatening and requires emergency medical treatment.
“Patients who are living with UCDs now have an alternative treatment option with Olpruva, to address some of the challenges they may have with existing therapy. We are pleased to be able to provide a new, approved treatment choice for those living with this challenging disease,” said Chris Schelling, CEO of Acer. “This approval represents the first FDA-approved product for Acer, validating our ability to identify and develop treatments where science can be applied in novel ways and make them available to patients as quickly and efficiently as possible.”
Olpruva received FDA approval under a regulatory pathway that allows applicants to rely, at least in part, on third party data for approval. In its New Drug Application, Acer cited preclinical and clinical safety and efficacy data from the reference listed drug, Buphenyl powder, which is approved as adjunctive therapy in the chronic management of patients with UCDs involving deficiencies of CPS, OTC or AS.
In its application, Acer also provided additional data including studies that evaluated the bioavailability and bioequivalence of Olpruva compared to Buphenyl powder. The data from these studies, presented at the Society for Inherited Metabolic Disorders Annual Meeting in April 2022 and the Genetic Metabolic Dieticians International Conference in May 2022, showed that Olpruva was bioequivalent to Buphenyl powder.
Author: Rare Daily Staff
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