The US Food and Drug Administration (FDA) has approved Acer Therapeutics’ olpruva (sodium phenylbutyrate) to treat patients with urea cycle disorders (UCDs).
Olpruva is a prescription medicine which has been approved as an oral suspension to treat UCD patients with deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).
The US FDA approved the medicine under section 505(b)(2) of the Federal Food, Drug and Cosmetic Act (FDCA).
The medicine is used along with certain therapy for the long-term management of UCDs in adult and paediatric patients.
Acer CEO and founder Chris Schelling said: “The FDA’s approval of olpruva, an innovative formulation of sodium phenylbutyrate packaged for the first time in single-dose envelopes, marks the culmination of our ongoing dedication to develop new and differentiated treatment options for those affected by rare diseases,”
“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.”
The company presented the preclinical and clinical data from the reference listed drug (RLD), buphenyl powder, which has been approved as adjunctive therapy of UCDs patients, in its New Drug Application (NDA).
It also provided the additional data including trials that assessed olpruva’s bioequivalence and bioavailability against buphenyl powder.
Acer stated that the regulatory approval of olpruva triggers a $42.5m of non-dilutive debt funding to the company under the previously announced loan agreement signed with Marathon Asset Management L.P affiliates.