WASHINGTON, DC — The Personalized Medicine Coalition released a new report documenting the U.S. Food and Drug Administration’s continued progress in advancing personalized medicine in 2025, including the approval of 16 new personalized medicines representing approximately 36% of all newly approved therapeutic molecular entities. The report also highlights the approval of five new gene- and cell-based therapies and significant advances in diagnostics, artificial intelligence, and rare disease innovation.

The personalized medicine approvals and policy developments at the FDA in 2025 demonstrate that scientific innovation continues to move healthcare away from one-size-fits-all medicine and toward more precise, patient-centered care,” said Gina Ross Murdoch, MBA, president and CEO of PMC. “These advances are helping improve outcomes, expand treatment options, and accelerate the integration of molecular information into clinical decision-making.”

• FDA approved 16 new personalized medicines, which have now accounted for more than one-third of new therapeutic approvals for each of the past six years.
• FDA approved five new gene- and cell-based therapies for patients with rare genetic diseases and hematologic cancers, including therapies for spinal muscular atrophy, Wiskott-Aldrich syndrome, recessive dystrophic epidermolysis bullosa, and idiopathic macular telangiectasia type 2.
• FDA expedited authorization of the first bespoke CRISPR gene-editing therapy developed for an individual patient with a rare metabolic disorder, signaling the emergence of highly individualized treatment approaches.
• FDA approved or cleared significant new and expanded indications for 16 companion diagnostic testing systems, including new blood-based biomarker tests for Alzheimer’s disease that support earlier and less invasive detection.
• FDA released draft guidance documents establishing frameworks for integrating artificial intelligence into drug development and medical devices and qualified the first AI-based drug development tool under its Drug Development Tool Qualification Program.
• FDA convened Rare Disease Innovation, Science and Exploration workshops to advance regulatory science and individualized therapies for patients with rare diseases.