Pathways & Standards

The chair of a European Medicines Agency working group on reducing animal testing said that companies can provide “thoughts and suggestions” in relation to a review of its scientific guidelines on regulatory testing requirements and new approach methodologies.

The former principal deputy commissioner said the “decapitation” of senior leadership will make resolving internal disputes more difficult, which will slow application reviews. Woodcock also called the FDA layoffs a slow-moving catastrophe.

A risk-based approach to human cell therapies and tissue-based products could incentivize development and prevent bad actors from taking advantage of the current FDA system.

US FDA Commissioner nominee Martin Makary is being embraced by industry, and Senate Democrats, as a more traditional pick than other Trump Administration nominees, but the Make America Healthy Again agenda still is clearly coming to the agency.

The former principal deputy commissioner said the “decapitation” of senior leadership will make resolving internal disputes more difficult, which will slow application reviews. Woodcock also called the FDA layoffs a slow-moving catastrophe.

The Brazilian medicines regulator will also offer more clarity on the requirements for radiopharmaceuticals that are exempt from registration.

Novartis is planning to file EU and US marketing applications for an intrathecal formulation of its spinal muscular atrophy gene therapy, Zolgensma, in H1 2025.

The FDA’s accelerated approval draft guidance has left stakeholders seeking clarification of the process for determining a surrogate marker or intermediate clinical endpoint is reasonably likely to confirm clinical benefit.

Massive layoffs and the biosimilar user fee program’s historic flirtation with the non-user fee spending requirements raise concerns that the FDA could lose its ability to collect BsUFA revenue, which could mean a fine or jail time for agency staff.

Mass FDA layoffs on 1 April were designed to spare product reviewers, but still touched many who are critical to the application review process or drug development, which could mean fewer treatments are brought to the US market first.

Sanofi’s Dupixent, Amgen’s Uplizna, and Bristol’s Opdivo seek new indications, while J&J hopes to start a franchise with nipocalimab and Stealth’s day of reckoning approaches.

FDA staffing cuts are making negotiation preparations more difficult, but also could mean the result is a smaller user fee renewal package.