FDA accepts re-submitted NDA for ACER 001 to treat patients with urea cycle disorders
Acer Therapeutics Inc. and its collaboration partner, Relief Therapeutics Holding SA announced the FDA has accepted for review Acer’s resubmitted New Drug Application (NDA) for ACER 001 (sodium phenylbutyrate) for oral suspension for the treatment of patients with urea cycle disorders (UCDs)
The FDA designated the NDA as a Class 2 resubmission and set a Prescription Drug User Fee Act (PDUFA) target action date of January 15, 2023.
In June 2022, as previously announced, the FDA issued Acer a Complete Response Letter (CRL) stating that satisfactory inspection of Acer’s third-party contract packaging manufacturer is required before the ACER 001 NDA may be approved. Acer notified the FDA in the NDA resubmission that the third-party contract packaging manufacturer is ready for inspection. The 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 required updates to the original NDA, were provided in the resubmission of the NDA.
Related news and insights
Roche announced that the FDA has approved a supplemental New Drug Application (sNDA) for Xofluza (baloxavir marboxil) for the treatment of acute uncomplicated influenza in otherwise healthy children aged five to less than 12 years of age who have been symptomatic for no more than 48 hours
AstraZeneca and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) has been approved in the US for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumours have activating HER2 (ERBB2) mutations, as detected by a FDA-approved test, and who have received a prior systemic therapy
The Menarini Group announced that the FDA has accepted the Company’s New Drug Application (NDA) for elacestrant, an investigational selective estrogen receptor degrader (SERD), for patients with ER+/HER2- advanced or metastatic breast cancer.