This past year, innovative biopharmaceutical companies received approval for 52 new medicines—46 new drugs and 6 new biologics and vaccines—demonstrating the industry’s commitment to advancing health for patients across a wide range of diseases. These new medicines reflect sustained private sector investment and a focus on addressing unmet medical needs.
Some of the key milestones for patients and biopharmaceutical researchers in 2025 included:
- Expanding the arsenal against cancer: Fourteen oncology approvals offer important new options for patients, including those with difficult-to-treat cancers. These advances include targeted therapies, which seek to improve outcomes by tailoring treatment to the specific genetic profile of a patient’s cancer. Oncology treatments contribute to increasing survival rates – which just reached 70% five-year survival rate for all cancers combined for U.S. patients diagnosed from 2015-2021.
- Innovations in rare diseases: Of the 46 novel drug approvals, 23 (50%) were designated by the U.S. Food and Drug Administration (FDA) as orphan (rare disease) drugs. One example from 2025 was the approval of a new treatment for hemophilia that can be administered less frequently than other treatment options.
- Advancing infectious disease treatment options: Noteworthy 2025 approvals included two oral medicines to treat gonorrhea – important because of the rise of gonococcal drug resistance globally.
- Improving quality of life for patients: Treatments that may significantly improve quality of life for patients facing chronic disease burden, pain or other symptoms impacting daily functioning can also benefit patients. One significant approval included a first-in-class non-opioid analgesic to treat moderate to severe acute pain, so patients have an option for acute pain relief instead of opioids.
Research doesn’t end when a medicine is approved.
When the FDA approves a novel treatment, the biopharmaceutical company’s work is not done. Researchers often continue studying these medicines to determine if they can be used to treat other diseases or whether they can be administered differently — like in a pill version instead of an injection — to help increase adherence and improve patients’ quality of life.
Additionally, the requirements under the Pediatric Research Equity Act (PREA) and incentives under the Best Pharmaceuticals for Children Act (BPCA) encourage continuing research into pediatric uses of approved medicines. For example, this past year, a drug initially approved in 2012 for use in adults was approved as the first treatment for irritable bowel syndrome with constipation for pediatric patients seven years and up.
American leadership
The U.S. continues to lead the world in the development and approval of new medicines, with 36 of the 52 (69%) novel medicines of 2025 approved first in the U.S. before other countries. The biopharmaceutical industry’s innovative drug research and development infrastructure plays a large role in this success. A predictable, science-based regulatory review by the FDA is also imperative. What’s more, legislation and programs the FDA has in place to efficiently review and approve new drugs, including the Prescription Drug User Fee Act (PDUFA) and expedited development and review pathways, help maintain America’s global competitiveness.
The range of approvals in 2025 signals promise for patient care and public health. Patients, clinicians and researchers alike will benefit as these therapies reach Americans and pave the way for new discoveries. Continued collaboration between regulators, industry, researchers, and patients is essential to sustain this momentum and maintain regulatory predictability so that America’s leadership in medicine remains strong and benefits every American.
With each year that passes, one thing remains steady: the biopharmaceutical industry is committed to advancing health for patients across a wide range of diseases. Because when people get sick, they may need a medicine.
Kristen Booze
Kristen Booze is a Director on PhRMA’s Public Affairs team, where she leads communications strategies on scientific and regulatory issues related to the biopharmaceutical industry. Prior to joining PhRMA, Kristen most recently served as Deputy Director of the Division of Health Communication in FDA’s Center for Drug Evaluation and Research (CDER). In this role, she advised on strategic communications and messaging related to drugs. Kristen served as a federal government communicator for twelve years. She holds a B.S. in biology from SUNY Geneseo and an MPH from The George Washington University. Outside of work, Kristen spends her time refereeing her three boys with her husband and squeezing in some early morning exercise.