In 2017, FDA’s Center for Drug Evaluation and Research’s (CDER’s) new drug therapy approvals helped a wide range of patients suffering from many different medical conditions—from rare disorders to common diseases—to gain new hope for improved quality of life, and in some cases, improved chances of surviving life-threatening illnesses.
Rare Diseases
Among many other new approvals to help patients with rare diseases, CDER approved the first new treatment for patients with sickle cell disease in almost 20 years and the first-ever non-blood product to treat patients with hemophilia A with inhibitors. For the first time, a treatment is available for adults diagnosed with giant cell arteritis, a rare condition that results in inflammation of blood vessels. CDER also approved a new treatment for the rare condition known as Batten disease, which can cause seizures, dementia, and a variety of other debilitating symptoms.
Infectious Diseases
We approved a new antibiotic to treat certain types of serious skin infections, and another to treat complicated urinary tract infections, including kidney infections. We also approved two new treatments for certain patients with chronic hepatitis C; a new drug to help prevent cytomegalovirus infection in patients who have received a bone marrow transplant; and, the first therapy in the United States to treat Chagas disease, a rare parasitic disease which, after years of infection, can cause serious heart illness.
Neurological Disorders
Last year was a particularly productive year for approving new therapies for patients with neurological disorders. CDER approved new therapies to treat patients with tardive dyskinesia, a frequent side effect of psychiatric medications, myasthenia gravis, a rare neuromuscular disease, and new treatments for Duchenne muscular dystrophy, for certain forms of multiple sclerosis, for amyotrophic lateral sclerosis (often called Lou Gehrig’s disease), and for Parkinson’s disease.
Cancer Therapies
2017 was also another strong year for making new cancer therapies available to patients in need. Among others, we approved new therapies for certain patients with acute lymphoblastic leukemia; Merkel cell carcinoma; certain forms of relapsed or refractory acute myeloid leukemia; certain forms of lymphoma; recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer; and specific forms of liver, breast, and colorectal cancer. We also approved the first cancer treatment based on a genetic feature of a cancer rather than the location of the body where the tumor originated.
Other Advances
Also in 2017, CDER approved a new therapy for decreasing heart risk for patients with diabetes, a new drug to treat adults with moderateto-severe eczema and three therapies to treat patients with moderateto-severe plaque psoriasis. Two of these were for adults and one was for adolescents. We also approved the first drug in the United States with a sensor embedded in the pill that records that the medication was taken. And in response to the devastation inflicted by the 2017 Hurricane Season, CDER worked with pharmaceutical manufacturers with facilities in affected areas to address potential drug shortages.
Written by
Janet Woodcock, M.D.
Director, Center for Drug Evaluation and Research, FDA
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