2026 Regulatory Momentum at the U.S. FDA

In 2025, the U.S. Food and Drug Administration (FDA) approved 58 brand new drugs, and while this is down from the prior year, this is still a remarkable feat given the U.S. political landscape and its impacts at the FDA. Innovation is still enormously present and will continue as we march into a new year. Some companies are restarting or picking up where they left off in 2025 to continue to bring new medicines to patients in need. 

Breakthroughs in HIV prevention, a non-opioid pain killer, new antibiotics, progress in cancer (notably within lung cancer), weight loss and weight management with GLP-1s made consistent headlines in 2025. However, upon reflection, Baby KJ’s medical miracle stands out the most – an N-of-1 treatment developed by the University of Pennsylvania in groundbreaking time resulting in a CRISPR (gene editing) treatment that corrected a genetic mutation within KJ’s liver allowing the six-month-old baby to avoid a liver transplant and resume a normal diet. While CRISPR has been on stage for the last three-to-five years, resulting in a Nobel prize in 2020 awarded to the researchers who developed this gene editing technology, this story has spawned further innovation within regulatory, some specific to N-of-1 treatments or ultra rare disease within the FDA.   

Some examples of new regulatory innovation from the FDA include: 

• The Agency introduced Rare Disease Evidence Principles (RDEP) in September 2025 to provide greater speed and predictability in the review of therapies intended to treat rare diseases with very small patient populations with significant unmet medical need driven by a known genetic defect. 

• The Agency’s new Plausible Mechanism Pathway outlined by Vinay Prasad, M.D., M.P.H and Martin A Makary, M.D., M.P.H. in the New England Journal of Medicine in November 2025. The FDA will reserve the use of this pathway for diseases for which the biological cause is known (i.e., genetic conditions with a clear connection between specific alteration and disease presentation), rather than diseases defined by a constellation of clinical findings or dozens of unclear genome wide associations, to safeguard against misapplication to disparate conditions with similar phenotypes. 

• The Commissioner’s National Priority Voucher Program (CNPV) was designed to accelerate the development and review of certain drugs and biological products aligned with U.S. national health priorities to enhance the health interests of Americans. The CNPV pilot program reduces drug and biological product application or efficacy supplement (ES) review times from 10-12 months to just one-to-two months. Announced in June 2025, this innovative program uses a collaborative board style review process to accelerate approvals for companies aligned with critical U.S. national health priorities. Despite this program receiving significant concerns from Ex-FDA leadership regarding politicalization and corruption risk, concerns on safety, given rushed reviews, lack of transparency, and risking public confidence, there have been 15 products approved under this program.  

• The Real-Time Release of Complete Response Letters (CRLs) issued to sponsors not only promotes transparency to the industry regarding the decisions concerning drug approvability but is one giant step forward in industry-wide knowledge sharing. This facilitates lessons learned and promotes more efficient drug development that saves both time and money, enabling quicker access to medicines.  

While some of these examples are still in their infancy, their impact on bringing new medicines to patients is considerable and should be added to your 2026 watch list.   

Despite the ever-changing leadership at the FDA, we witnessed throughout 2025 (and at Halloran) regulatory review teams’ resilience and commitment to protecting the public. They remain engaged, and despite having far fewer team members, they provided meaningful feedback to meet procedural timelines for many open applications. 

As our industry continues to innovate by bringing forward generics and biosimilars, addressing the impending patent cliff through intense deal making and M&A activity, refinement and enhancement of first generation of drugs, further progressing advanced therapies, charting into uncharted territories of medicine in collaboration with technology and AI, we must rely on the FDA’s resilience and partner with them to bring these innovations to patients. 

It would be naïve to think there are no obstacles ahead of us, however, there should be optimism, albeit cautious optimism for 2026. There are expectations of rebound and healthier market performance to derisk investment, which is the lifeblood of the biopharma industry.  There is momentum with regulatory policy reforms and flexibility to promote continued partnership and dedication to bringing new medicines to patients.  

With JPM and the Biotech Showcase just ahead of us, the 2026 outlook haze within the crystal ball will likely come into better focus. Excitement is in the air as we embark on a new chapter. Attending JPM and/or the Biotech Showcase? Contact us today. We’d love to connect onsite.