Issued January 1, 2023
Issued
January 1, 2023
Application
Other • 214860
Review center
Other
Stage
Final Decision
Letter type
Approval Letter
This is a Complete Response letter from the FDA to Acer Therapeutics Inc. regarding their New Drug Application (NDA) 214860 for sodium phenylbutyrate. The FDA has determined that the application cannot be approved in its current form due to several deficiencies, including an incomplete facility inspection, issues with prescribing information and labeling, and requirements for a safety update and additional nonclinical data.
The application cannot be approved in its present form due to an uncompleted facility inspection, multiple deficiencies in prescribing information and carton/container labeling, and the need for a comprehensive safety update. Additionally, the proprietary name needs resubmission, and further information is required regarding a nonclinical testicular weight finding.
The field investigator could not complete the inspection of Sharp Clinical Services, Inc. manufacturing facility (FEI# 3003673570) because the facility was not ready for inspection. A satisfactory inspection is required for approval.
Recommended response: Ensure the manufacturing facility is fully prepared for inspection and promptly notify the agency in writing when it is ready.
Draft labeling is not responsive to the electronic communication dated June 15, 2022. Formatting errors need correction using the SRPI checklist. An updated content of labeling in Structured Product Labeling (SPL) format is required, conforming to 21 CFR 314.50(l)(1)(i), 201.56(a), 201.56(d), and 201.57.
Recommended response: Revise the Prescribing Information according to FDA's electronic communication, the SRPI checklist, and relevant regulations. Submit in SPL format, including both a marked-up and a clean Word version.
Cited: 21 CFR 314.50(l)(1)(i), 21 CFR 201.56(a), 21 CFR 201.56(d), 21 CFR 201.57
Draft carton and container labeling must be submitted based on the proposed revisions and communications dated May 24, 2022, and May 31, 2022.
Recommended response: Submit revised carton and container labeling as per FDA's previous communications.
The proposed proprietary name, Olpruva, was found acceptable pending approval but needs to be resubmitted when responding to the application deficiencies.
Recommended response: Resubmit the proposed proprietary name 'Olpruva' along with the complete response to other deficiencies.
A comprehensive safety update is required as described at 21 CFR 314.50(d)(5)(vi)(b). This includes data from all nonclinical and clinical studies, significant changes in the safety profile, updated adverse event tabulations, case reports for deaths/serious AEs, updated exposure information, and a summary of worldwide experience.
Recommended response: Provide a detailed safety update according to 21 CFR 314.50(d)(5)(vi)(b), incorporating all new safety data, updated analyses, and worldwide experience.
Cited: 21 CFR 314.50(d)(5)(vi)(b)
In the chronic dog study of talc, absolute testis weights were statistically-significantly increased, and no NOAEL was established. Additional information (exact ages of dogs, review of published studies on talc) is requested to assess this finding. A new 9-month study may be needed if clarification is insufficient.
Recommended response: Provide the requested information on dog ages and published talc studies. Be prepared to conduct a new 9-month dog study if the finding remains unclear after initial assessment.
The application received a Complete Response due to critical manufacturing site readiness issues, significant deficiencies in labeling content and format, and the need for a comprehensive update to the safety profile. Addressing these core issues is essential for future approval.
Regulatory change impact: Pending sponsor mitigation plan
Approval likelihood after response: 5%
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