PhRMA Reports 450 Medicines in Development for Rare Diseases

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.

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