Article

PhRMA Reports 450 Medicines in Development for Rare Diseases

Author(s):

PhRMA reports 452 new medicines are in development for rare diseases, including genetic disorders, neurological conditions, infectious diseases, and autoimmune disorders.

The

(PhRMA) reports that biopharmaceutical research companies are developing 452 new medicines for rare diseases, including genetic disorders, neurological conditions, infectious diseases, and autoimmune disorders. According to FDA, in the last five years, one third of all new drug approvals were for rare diseases.

The 452 medicines and vaccines in development for rare diseases, all in human clinical trials or under review by FDA, include 105 for cancer, 85 for genetic disorders, 65 for blood cancers, and 32 for neurological disorders.

Source:

Newsletter

Stay at the forefront of biopharmaceutical innovation—subscribe to BioPharm International for expert insights on drug development, manufacturing, compliance, and more.

Related Videos
Behind the Headlines, Episode 21: Waters-BD Merger, Merck’s $10B Bet, and Biotech’s Investment Frontiers
© 2025 MJH Life Sciences

All rights reserved.