SOUTH SAN FRANCISCO (dpa-AFX) - The FDA's October decision calendar looks relatively light, with only a limited number of PDUFA dates scheduled.
As we turn to October, here's a recap of some of the regulatory developments that made headlines in September.
The month saw several notable FDA milestones, including approvals that marked important firsts in the treatment of rare diseases.
Ionis Pharmaceuticals Inc.'s (IONS) Zanvastro received FDA approval on September 3, becoming the first drug approved to treat Alexander disease, a rare neurological disorder.
On September 17, the FDA approved Ultragenyx Pharmaceutical Inc.'s (RARE) gene therapy Fayuvi as the first treatment for pediatric patients with Sanfilippo syndrome type A, a rare, progressive neurodevelopmental disease.
Egetis Therapeutics US Inc.'s Emcitate was approved on September 28, becoming the first treatment for MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome, a very rare genetic disorder.
So far this year, from January through September 2026, 44 novel drugs have received FDA approval, compared with 32 novel drugs approved during the same period in 2025.
The U.S. regulatory agency, currently headed by Acting Commissioner Kyle Diamantas, J.D., is also preparing for a change in leadership. Marty Makary resigned as FDA Commissioner in May 2026 after roughly 13 months in office amid disputes over several FDA policy issues.
Against this backdrop, on September 24, Heidi Overton, President Donald Trump's nominee to lead the FDA, appeared before the Senate Health, Education, Labor, and Pensions (HELP) Committee for her confirmation hearing. She faced questions on a range of issues, including the abortion pill mifepristone, the safety of the measles, mumps, and rubella (MMR) vaccine, and food safety.
If confirmed, Overton, 37, would become the youngest FDA commissioner in the agency's history and its 28th Commissioner of Food and Drugs.
Now, let's take a look at the biotech stocks facing FDA decision in October.
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