By Mary de Wet


Acer Therapeutics Inc. has received approval from the Food and Drug Administration for Olpruva, the first of four drugs the company was investigating.

The drug, also known as sodium phenylbutyrate and ACER-001, is approved as adjunctive therapy for adult and pediatric patients with certain urea-cycle disorders.

The FDA deferred submission of Acer's study of pediatric patients whose body weights fall outside of the parameters granted by the approval, according to a letter by the agency dated Thursday.

Studies to develop appropriate dosage strengths for patients who weigh less than 20 kilograms or who weigh more than 20 kilograms with a body surface area greater than 1.2 square meters are necessary to ensure appropriate dosing of Olpruva in these patients, the FDA said in the letter.

"The development of such dosage strength(s) will depend upon the feasibility of manufacturing and demonstration of stability of lower strength(s)," the agency said in the letter.

Acer has a collaboration and license agreement with Relief Therapeutics Holding AG for Olpruva. The European Commission in August granted orphan medicinal product designation to the drug for the potential treatment of patients with Maple Syrup Urine Disease in the EU.

Acer's development pipeline comprises Olpruva, ACER-801 to treat induced vasomotor symptoms, Edsivo to treat vascular Ehlers-Danlos syndrome in patients with a type III collagen mutation, and ACER-2820 to treat a variety of viruses, including Covid-19 and Ebola.


Write to Mary de Wet at mary.dewet@dowjones.com

(END) Dow Jones Newswires

12-23-22 1550ET