US legislation could advance new manufacturing platforms for drugs and biologics with academic R&D and workforce development and in turn spur domestic growth.
Legislation promoting the research and development of advanced and continuous pharmaceutical manufacturing technologies has again won US House approval, this time as part of must-pass FDA user fee legislation, which should increase its chances of finally winning Senate approval and becoming law.
The advanced and continuous manufacturing provision is missing from the version of the legislation to renew user fees for another...
Many platform designation requests have been from sponsors eager to cite other sponsors’ products, but CBER Director Peter Marks said in an interview with the Pink Sheet his office likely is years away from accepting those applications.
CBER Director Peter Marks outlines a streamlined process to approval for treating different mutations of the same gene. NCATS’ Philip Brooks tells the Pink Sheet the approach avoids the need to “start from scratch for every new mutation.”
New draft guidance defines the necessary components of a platform technology designation request, including that sponsors demonstrate how it will save the FDA review time.
CBER’s Nicole Verdun said the FDA is using the principles of the platform approach in situations that don’t qualify for the agency’s new incentive.
Long-term follow-up requirements have taken a conservative approach but could be ripe for re-examination and global harmonization given the years of experience with the products, Kite Pharma executive director says; former FDA gene/cell therapy office head Wilson Bryan calls for elimination of the classwide REMS.
Program for collaborative reviews of gene therapy applications is getting under way with the European Medicines Agency. After launch, the pilot could be expanded to earlier discussions around CMC and pharm/tox data requirements, FDA’s Nicole Verdun said.
