Acer and Relief win approval for Olpruva following inspection issues

Acer Therapeutics and its Swiss partner Relief Therapeutics celebrated an FDA approval on Tuesday for Olpruva, an oral drug for urea cycle disorders (UCDs).

The news comes roughly six months after regulators rejected Olpruva, also known as ACER-001, because its contract packaging manufacturer wasn’t ready for an inspection, according to the partners. The FDA requested “additional existing nonclinical information,” but the companies said it was “not an approvability issue.”

They refiled a new drug application in July, just one month after receiving the complete response letter (CRL).

“Our third-party contract manufacturing partner has been incredibly responsive and has confirmed that it is ready for inspection,” Acer CEO Chris Schelling said at the time. “Our manufacturing partner is regarded as a global leader in clinical supply chain and commercial packaging services with more than 70 years of experience.”

Urea cycle disorder is a rare genetic condition in which ammonia builds up in the blood. Elevated ammonia levels in the blood can lead to swelling of the brain, and eventually cognitive impairment or death.

Jack Weinstein

Olpruva, an oral formulation of sodium phenylbutyrate, is now approved to treat certain patients with UCDs that involve deficiencies of carbamyl phosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). It’s designed to minimize the unpleasant taste that Acer said has led to compliance issues with other drugs containing the compound.

Olpruva marks Acer’s first FDA approval. It also allows the company access to a previously announced $42.5 million term loan from Marathon Asset Management that was contingent upon the approval.

“This FDA approval is a significant milestone for patients with UCDs in the U.S., offering an additional choice to manage their condition,” Relief CEO Jack Weinstein said in a news release.  “We look forward to building on OLPRUVA’s approval in the U.S. and expanding its availability into other territories outside of the U.S.”

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