Acer Therapeutics Inc. (NASDAQ:ACER) (" Acer "), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, and RELIEF THERAPEUTICS Holding AG (SIX: RLF, OTCQB: RLFTF) (" Relief "), a biopharmaceutical company seeking to provide patients therapeutic relief from serious diseases with high unmet need, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for ACER-001 (sodium phenylbutyrate) for the treatment of Urea Cycle Disorders (UCDs). ACER-001 is a nitrogen-binding agent in development for use as adjunctive therapy in the chronic management of patients with UCDs involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).
Based on standard FDA review timelines, Acer expects to receive notification from FDA on the potential acceptance of the NDA for filing within 60 days of submission and subsequent substantive review.
The 505(b)(2) NDA submission is supported by results from two previously announced bioequivalence (BE) trials in which ACER-001 showed similar relative bioavailability for both phenylbutyrate (PBA) and phenylacetate (PAA), the active metabolite of sodium phenylbutyrate, compared to BUPHENYL(R) (sodium phenylbutyrate). Acer has also received an Agreement Letter from FDA in response to the Company's initial Pediatric Study Plan (iPSP) which outlines an agreed upon approach that addresses the needs of pediatric patients with UCDs.
"The submission of our NDA for ACER-001 marks an important step toward bringing this potential alternative treatment option to patients with UCDs," said Chris Schelling, Chief Executive Officer and Founder of Acer . "We look forward to working with the FDA through the review process and will continue advancing our preparations for a potential launch of ACER-001, while also assisting Relief toward regulatory submissions in Europe."
Jack Weinstein, Chief Financial Officer and Treasurer of Relief Therapeutics , added, "We are excited about the progress made to date in support of a potential regulatory approval of ACER-001 for UCDs in the U.S. With the NDA now submitted, we will continue our efforts to back ACER-001's development in Europe by targeting submission of a Marketing Authorization Application (MAA) for the treatment of UCDs in Europe by the end of 2021."
ACER-001 is an investigational product candidate which has not been approved by FDA or the European Medicines Agency (EMA). There is no guarantee that this product candidate will be accepted for substantive review, or if accepted, receive regulatory authority approval in any territory, or become commercially available for the indications under investigation. 2021 Global Data Point.