The U.S. Food and Drug Administration announced the global launch of CURE ID, an internet-based repository that will allow the clinical community to report their experiences treating difficult-to-treat infectious diseases with novel uses of existing FDA-approved drugs through a website, a smartphone or other mobile device. The U.S. Food and Drug Administration has approved three applications for first generics of Gilenya (fingolimod) capsules for the treatment of relapsing forms of multiple sclerosis (MS) in adult patients.
MS is a chronic, inflammatory, autoimmune disease of the central nervous system that disrupts communication between the brain and other parts of the body.
Today, the U.S. Food and Drug Administration granted accelerated approval to Oxbryta (voxelotor) for the treatment of sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older.
"Today's approval provides additional hope to the 100,000 people in the U.S., and the more than 20 million globally, who live with this debilitating blood disorder," said Acting FDA Commissioner Adm. Brett P. Giroir, M.D.
Today, the U.S. Food and Drug Administration granted approval to Givlaari (givosiran) for the treatment of adult patients with acute hepatic porphyria, a genetic disorder resulting in the buildup of toxic porphyrin molecules which are formed during the production of heme (which helps bind oxygen in the blood). Today, the U.S. Food and Drug Administration approved Adakveo (crizanlizumab-tmca), a treatment to reduce the frequency of vaso-occlusive crisis - a common and painful complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells - for patients age 16 years and older. Today the U.S. Food and Drug Administration granted approval to Reblozyl (luspatercept-aamt) for the treatment of anemia (lack of red blood cells) in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions. The U.S. Food and Drug Administration is taking steps towards requiring electronic submission of certain safety reports for products being evaluated by the FDA under an investigational new drug (IND) application into the FDA's Adverse Event Reporting System (FAERS). The planned process for submissions is outlined in a new draft guidance, and the FDA is also making available supporting technical specification documents.