Pathways & Standards
Approval Standards
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 Pink Sheet's list of EU centralized approvals of new active substances has been updated to include four new products, one of which is Vimkunya, Bavarian Nordic's chikungunya vaccine.
The FDA generic drugs team’s first public workshop of the second Trump Administration ended with a request that industry amplify the value it finds from public engagement.
Review Pathways
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The FDA’s accelerated approval draft guidance has left stakeholders seeking clarification of the process for determining a surrogate marker or intermediate clinical endpoint is reasonably likely to confirm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.
Experts working in the advanced therapy space say the US has less strict criteria for regulatory pathways for cell and gene therapies than the EU, particularly for products in early development.
User Fees
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.
Republican congressional staffers could bring a wealth of experience and strong contacts with key legislators to the FDA ahead of the next user fee reauthorization process. But will Health and Human Services Department leadership interfere?