2017: A Year of Innovation and Advances 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