Resubmission of NDA for ACER 001 for treatment of urea cycle disorders.

Acer believes the resubmission addresses in full the items raised by the FDA in the Complete Response Letter (CRL).

In June 2022, as previously announced, the FDA issued Acer a CRL stating that satisfactory inspection of its third-party contract packaging manufacturer is required before the ACER 001 NDA may be approved. Acer notified the FDA in the resubmission that the third-party contract packaging manufacturer is ready for inspection. FDA did not cite any other approvability issues in the CRL pertaining to the NDA, nor request any additional clinical or pharmacokinetic studies be conducted prior to FDA action. Additional existing nonclinical information as requested by the FDA in the CRL but identified as “not an approvability issue”, as well as labeling and other routine updates to the original NDA, were provided in the resubmission of the NDA.

Acer expects to be notified by the Agency of its decision to accept or reject the resubmission for review within 14 calendar days of receipt of the NDA resubmission. If the resubmission is deemed complete by the FDA, a resubmission classification (Class 1 or 2) will be assigned and a new Prescription Drug User Fee Act (PDUFA) target action date will be established of either two or six months from the resubmission date depending on the classification and an inspection of the facility will be requested.