Scientific, political pressures colliding at FDA, delaying rare disease treatment for patients who have few or no options, experts said at CNBC Cures Summit.

In a major shift, the US Food and Drug Administration is relaxing a long-standing drug approval requirement for common diseases.

A panel of physicians, biotech leaders and patient advocates took aim at the FDA during a Senate hearing Thursday on how the ...

A Senate hearing probed whether the Food and Drug Administration's process for approving rare-disease therapies is ...

The FDA proposed new guidelines for approving experimental therapies for rare diseases without large group studies.

When the US Food and Drug Administration (FDA) approved benzgalantamine (Zunveyl) in July 2024 for Alzheimer’s disease, it was hailed by its manufacturer as a ‘major breakthrough’.1 In reality, ...

Here’s how drug developers can best approach interactions with the agency following last year’s seismic changes to its leadership, workforce and policies.

“Designing treatments unique to individual patients has always been the promised goal of personalized medicine. After 25 years the FDA has, for the first time, outlined a framework to facilitate these ...

Once the active ingredient is defined, an Applicant must select which patent, or patents, to put forward for extension. The statute permits only one PTE per regulatory review period, so the choice is ...

In an interview, C. Ola Landgren, MD, PhD, delved into the details of the FDA's draft guidance on using MRD as a basis for ...

Against the backdrop of Rare Disease Day advocates visiting Capitol Hill this week, a Senate panel raised alarm on Thursday ...

Investing.com -- UniQure N.V. (NASDAQ:QURE) shares fell 22% Thursday after FDA Commissioner Marty Makary made comments about a rare disease drug trial that appeared to reference the company’s ...