The following is an excerpt from a blog post by Nicole (Niki) Gallo, Vice President of Regulatory Affairs at Halloran, a PLG Company.
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.