Ligand And GlaxoSmithKline Receive FDA Breakthrough Therapy Designation For Promacta
By Cyndi Root
Ligand Pharmaceuticals announced in a press release that its partner, GlaxoSmithKline, received Food and Drug Administration (FDA) breakthrough therapy designation for Promacta. The drug treats severe aplastic anemia (SAA) in those who have not responded to immunosuppressive therapy. John Higgins, President and CEO of Ligand said, “We congratulate the global Promacta/Revolade development team at GSK, and commend them for their continued scientific and regulatory progress.”
SAA
Severe Aplastic Anemia is a rare bone marrow disorder whereby the marrow does not make enough blood cells. Bone marrow, the spongy tissue in the center of the bones, makes stem cells, which turn to blood cells: red, white, and platelets. The red blood cells carry oxygen and carbon dioxide. Their life cycle is about 120 days, necessitating the manufacture of new cells by the bone marrow. When the marrow does not make enough blood cells, the body responds with several types of disease states. Patients experience enlarged hearts, arrhythmias, bleeding, infections, and even death. People who do not respond to immunosuppressive therapy (IST) currently have no approved therapies. Almost half of these patients die within five years from bleeding or infection.
Promacta
Promacta is already FDA approved for thrombocytopenia in patients with chronic hepatitis C and for thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. It has some side effects including liver problems, jaundice, fatigue, stomach pain or swelling, and mental confusion. Liver problems may increase if the patient has the hepatitis C virus with cirrhosis or the patient takes Promacta with interferon and ribavirin.
Breakthrough Therapy Designation
In 2012, the FDA enacted the FDA Safety and Innovation Act (FDASIA), which included the Breakthrough Therapy designation. The designation expedites drug development by more quickly reviewing drugs that have potential to treat serious and life-threatening diseases or conditions. FDA reviewers considered the results from a National Institute of Health (NIH) study on Promacta. The open-label Phase II study (09-H-0154) included 43 SAA patients who had failed to respond to IST.
Ligand Pharmaceuticals acquires and licenses pharmaceuticals for a wide range of conditions including diabetes, hepatitis, and osteoporosis. The company has multiple partnerships with leading drug companies like GlaxoSmithKline.
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