Drug Approval Standards

Requests for “enforcement actions are not within the scope of FDA’s citizen petition procedures,” CDER says, rejecting petition dosing device firm Parenteral Technologies submitted as it prepares for workshop on Pediatric Research Equity Act requirements for OTC NDA sponsors.

The FDA’s vaccine advisory committee endorsed a new plan to update pandemic influenza vaccines “inter-pandemic,” but encouraged the agency to consider how to make the approach feasible for novel vaccine technologies.  

NDAs for additional OTC products containing acetaminophen and/or NSAIDs and indicated for use by children between 2 and less than 12 years old would trigger compliance by application sponsors with the act passed in 2003 to address lack of pediatric use information in drug labeling.

Dalzanemdor missed its primary endpoint in a Phase II Parkinson’s study, Sage announced, following April’s Phase II miss in Alzheimer’s. Phase II data in Huntington’s are still expected this year.

Phase III success positions Scholar Rock’s selective myostatin inhibitor for regulatory filings in early 2025. Analysts see a blockbuster opportunity for additive therapy in spinal muscular atrophy.

Regenerative Processing replaces nozzle to prevent backflow for its Regener-Eyes drops but FDA warning states numerous questions about sterility at the firm’s plant and about its procedures and systems for preventing microbial contamination.

Avidity hopes to complete enrollment in mid-2025 for an ongoing Phase III trial of its antibody-oligonucleotide conjugate in myotonic dystrophy type 1, which has no approved drug therapy.

Brazil has also published its recent regulatory reliance regulations in English, a move designed to increase confidence in its decisions as it presses on with aligning its national practices with global best practices.

Nonbinding forecast, fourth for the program, includes risks associated with codeine-containing cough medicine as a topic FDA will include in ongoing evaluation of GRASE for pediatric cough cold drug products marketed under monograph for cold, cough, allergy, bronchodilator and anti-asthmatic OTC drugs.

The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads.