On Jan. 14, 2025, the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) announced it had approved 50 new drugs never before approved or marketed in the U.S., known as “novel” drugs in 2024.

CDER’s 2024 drug approvals included therapies for a variety of diseases and conditions, including rare diseases. The FDA approved a significant number of rare disease therapies with orphan drug designation for patients with few to no treatment options. Therapies with orphan drug designations accounted for over 50% of our novel drug approvals, highlighting our steadfast commitment to address the huge unmet medical need in rare diseases.

The FDA approved 26 novel therapies in 2024 with this designation, including two of which treat conditions with no previously approved treatment such as Neimann-Pick disease type C and WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis).