Ann: Type C Meeting granted by FDA for Phelan-McDermid syndrome, page-78

• Dr. Patrizia Cavazzoni, leading CDER, was pivotal in drug evaluation and approval processes, handling new and generic drug applications. Her resignation on January 18, 2025, as reported by BioPharma Dive, could delay drug review timelines, especially for novel therapies.
• Dr. Peter Marks, heading CBER, managed vaccines, gene therapies, and blood products. His forced resignation, effective April 5, 2025, as detailed by STAT News, raises concerns about vaccine safety oversight, particularly amid ongoing measles outbreaks and vaccine hesitancy, as noted in his resignation letter criticizing HHS Secretary Kennedy.

Neuren Pharmaceuticals (ASX:NEU), focused on neurodevelopmental disorder therapies, notably trofinetide (DAYBUE) for Rett syndrome, already holds FDA approval. The company’s interactions are primarily with CDER for drug approvals, which would be directly affected by Dr. Cavazzoni’s resignation on 18 January 2025.

The Type C meeting (was granted on 6 February 2025) with FDA scheduled in early April, was granted after Dr. Cavazzoni’s resignation.
Also Soleno new drug approval has not been impacted by the staff change.


Key Points

• Research suggests the new CDER leader is Jacqueline Corrigan-Curay, acting director, following Dr. Patrizia Cavazzoni's resignation.
• It seems likely that FDA staff changes had minimal impact on Soleno Therapeutics' drug approval, with Vykat XR approved on March 26, 2025, despite a review extension.
• The evidence leans toward no current delay for Soleno, as their drug was approved, but past delays may have occurred during the review process.

New Leader of CDER

The current acting director of the Center for Drug Evaluation and Research (CDER) is Jacqueline Corrigan-Curay, J.D., M.D., who took over after Dr. Patrizia Cavazzoni resigned on January 18, 2025. This leadership change reflects ongoing transitions within the FDA, particularly under the new administration.
Impact on Soleno Therapeutics

Soleno Therapeutics' drug, Vykat XR (diazoxide choline extended-release tablets), was approved by the FDA on March 26, 2025, for treating hyperphagia in Prader-Willi syndrome. Despite an earlier extension of the review period to March 27, 2025, from December 27, 2024, the drug was approved, suggesting minimal impact from staff changes. While it's possible leadership transitions, including Cavazzoni's resignation in January 2025, contributed to past delays, no direct evidence links these changes to the timeline, and the approval proceeded as expected.