In 2025, the FDA approved 46 novel drugs, with 26 (57%) designated as orphan treatments for rare diseases.9
On February 23, 2026, the FDA launched the 'Plausible Mechanism Framework' to accelerate approvals for ultra-rare diseases by allowing plausible mechanisms to bypass certain clinical trial requirements.68
In September 2025, the FDA introduced the Rare Disease Evidence Principles (RDEP) to speed up genetic therapy reviews for rare diseases.3
Recent 2025-2026 approvals include Kygevvi for thymidine kinase 2 deficiency (Nov 3, 2025), Zycubo for Menkes Disease (Jan 12, 2026), and Forzinity for Barth Syndrome (Sep 19, 2025).2
Multiple orphan drugs received PDUFA approvals in late 2025, such as Plozasiran for familial chylomicronemia syndrome (Nov 18, 2025).1
Sources:
1. https://checkrare.com/2025-orphan-drugs-pdufa-dates-and-fda-approvals/
2. https://www.fda.gov/about-fda/accelerating-rare-disease-cures-arc-program/rare-disease-drug-approvals
3. https://www.definitivehc.com/blog/rare-disease-news
6. https://www.jdsupra.com/legalnews/fda-announces-new-approval-pathway-for-7786795/
8. https://www.hhs.gov/press-room/fda-launches-framework-accelerating-development-individualized-therapies-ultra-rare-diseases.html