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

The FDA guidelines aim to create a new pathway for bespoke therapies due to the challenges of conducting large studies.

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

FDA Will Drop Two-Study Requirement for New Drug Approvals, Aiming to Speed Access U.S News February 18, 2026 ...

This move is another step in Makary’s attempts to shorten FDA reviews, which started when he began his tenure last year. 3 These include mandating the use of artificial intelligence for staffers and ...

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.

After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...