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Orphan drugs account for more than half of all FDA drug approvals in 2020

In 2020, more than half (31 of 53, or 58%), of all of novel approvals rendered by the FDA were for drugs to treat patients with rare diseases. Among many others, these diseases include:

  • Spinal muscular atrophy, Duchenne muscular dystrophy, tuberous sclerosis complex, Dravet syndrome, neuromyelitis optica spectrum disorder, lupus nephritis, Still’s disease, hypereosinophilic syndrome, pro-opiomelanocortin deficiency, Cushing’s disease, thyroid eye disease, Ebola, and severe malaria;

  • Rare cancers including, multiple myeloma, high-risk refractory or relapsed neuroblastoma, gastrointestinal stromal tumor, epithelioid sarcoma, metastatic cholangiocarcinoma, and mesothelioma.

Read about all FDA approvals, including new and expanded uses of previously approved products, and biosimilars, in this newly published booklet available at


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