An experimental product candidate, ACER-001 is being developed to treat several inborn errors of metabolism, such as UCDs and maple syrup urine disease (MSUD).
The application to the FDA is supported by already reported data from two bioequivalence clinical trials of ACER-001.
In the trials, the product demonstrated comparable relative bioavailability for phenylbutyrate (PBA) as well as phenylacetate (PAA) versus the reference listed therapy, Buphenyl (sodium phenylbutyrate).
PBA and PAA are the active sodium phenylbutyrate metabolites, Acer noted.
The FDA has set the Prescription Drug User Fee Act (PDUFA) target action date to 5 June next year.
Acer Therapeutics CEO and founder Chris Schelling said: “With FDA commencing a substantive review of our NDA, ACER-001 is one step closer to potentially providing an alternative treatment option for patients with UCDs.
“In addition, we continue to collaborate with our partners to ensure we are well-positioned to support a successful commercial launch of ACER-001, subject to FDA approval.”
A group of ailments caused by genetic mutations, UCDs lead to deficiency in one of the six enzymes that catalyse the urea cycle.
This, in turn, can cause excessive ammonia build-up in the bloodstream, a condition called hyperammonemia.
Relief Therapeutics chief financial officer and treasurer Jack Weinstein said: “Our collaboration with Acer is thriving and we are pleased with the progress they have made in advancing ACER-001.
“In parallel with Acer’s activities, we continue to execute on our global commercial strategy for ACER-001 which includes our intended submission of a Marketing Authorization Application (MAA) for the treatment of patients with UCDs in Europe in Q2/Q3 2022.”