As BsUFA IV negotiations move forward, sponsors should expect renewed focus on review efficiency, evidentiary expectations, and policy alignment. Regulatory strategy, evidence planning, and early engagement with FDA will be critical as potential changes take shape.
As therapies become more complex, early regulatory engagement is no longer a best practice but a necessity. Cell and gene therapies, combination products, and novel biologics often challenge traditional regulatory frameworks, making late-stage alignment with regulators increasingly risky.
The International Council for Harmonisation (ICH) is signaling a significant shift in how global regulatory guidelines address cell and gene therapies, reflecting the growing complexity and rapid evolution of these advanced products. The ICH Cell and Gene Therapy Discussion Group (CGTDG) has released a discussion paper recommending a more tailored, risk-based approach to regulation that better reflects the unique scientific and manufacturing characteristics of cell and gene therapy products compared to traditional pharmaceuticals.
A newly proposed draft guidance on the development of regenerative medicine therapies has prompted significant feedback from industry groups, research organizations, and professional associations. While stakeholders broadly support efforts to strengthen pathways for advanced therapies, many are asking for greater clarity on eligibility criteria for the Regenerative Medicine Advanced Therapy (RMAT) designation and how it should be applied in practice.
The approval pathway for CAR T-cell therapies is entering a new phase as regulators signal a shift toward more rigorous clinical evidence. Leaders from the Center for Biologics Evaluation and Research (CBER) outlined an updated regulatory approach in a recent JAMA publication, indicating that randomized controlled trials (RCTs) will become the preferred standard for most future CAR T approvals.
At the 2025 Food and Drug Law Institute (FDLI) Enforcement, Litigation, and Compliance Conference, senior leadership from the FDA’s Center for Biologics Evaluation and Research (CBER) emphasized that sponsors must be fully prepared for preapproval inspections (PAIs) much earlier in the review cycle than many currently plan for. Melissa Mendoza, Director of the Office of Compliance and Biologics Quality (OCBQ), warned that delaying inspection readiness can jeopardize an application and compress the time needed to resolve inspection findings.
A newly proposed draft guidance on the development of regenerative medicine therapies has prompted significant feedback from industry groups, research organizations, and professional associations. While stakeholders broadly support efforts to strengthen pathways for advanced therapies, many are asking for greater clarity on eligibility criteria for the Regenerative Medicine Advanced Therapy (RMAT) designation and how it should be applied in practice.
The U.S. Food and Drug Administration (FDA) has released new draft guidance that could significantly reduce the reliance on non-human primates in safety testing for monospecific monoclonal antibodies. The document, Monoclonal Antibodies: Streamlined Nonclinical Safety Studies, outlines ways sponsors can assess long-term safety using shorter studies, alternative models, or in some cases no animal testing at all.
The US Food and Drug Administration (FDA) is receiving significant feedback from industry, patient groups, and academic organizations following the release of its updated draft guidance on safety labeling changes (SLCs). Commenters are seeking more clarity on how the agency identifies new safety signals, how labeling decisions are made, and what information FDA will share publicly throughout the process.
The U.S. Food and Drug Administration (FDA) has appointed Dr. Richard Pazdur as the new Director of the Center for Drug Evaluation and Research (CDER), following the abrupt resignation of former director George Tidmarsh. The transition marks a major leadership shift for the agency’s largest center, which oversees the regulation of prescription drugs, generics, and therapeutic biologics.