The FDA’s Center for Drug Evaluation and Research (CDER) released the annual report, “Advancing Health Through Innovation:
New Drug Therapy Approvals”, outlining new drugs approved for the first time.
Included in the report were numerous approvals for treatments targeted at combatting rare diseases like cystic fibrosis, tenosynovial giant cell tumor, and sickle cell disease. Additional approvals were given to assist patients with erythropoietic protoporphyria, neuromyelitis optica spectrum disorder, systemic sclerosis-associated interstitial lung disease, and Duchenne muscular dystrophy. Because so few patients suffer from these diseases, there are often scarce or no drugs available to treat the conditions.