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 ma
