FDA Approves Rigel’s Treatment for Chronic Immune Thrombocytopenia (ITP) in Adult Patients

Rare Daily Staff

The U.S. Food and Drug Administration approved Rigel Pharmaceuticals’ Tavalisse for the treatment of adults with chronic immune thrombocytopenia, a rare disorder in which the body’s immune system attacks the body’s own blood platelets.

Common symptoms of ITP include excessive bruising, bleeding and fatigue. People suffering with chronic ITP may face an increased risk of severe bleeding that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters and splenectomy, although they are not adequate for all patients.

The FDA approval Tavalisse, also known as fostamatinib, is a first for Rigel. Its use is approved for patients who have an insufficient response to a previous treatment. The drug is designed to inhibit SYK, a key signaling component in the body’s immune process that can lead to platelet destruction in ITP patients and may address an underlying of the condition.

Raul Rodriguez, president and CEO of Rigel, said the approval “validates the therapeutic effect of SYK inhibition in an autoimmune disease.”

“Chronic ITP is challenging to treat because the heterogeneity of the disease makes it difficult to predict how an individual patient will respond to available treatments and not all patients can find a treatment that works well for them,” said James Bussel, professor emeritus of pediatrics at Weill Cornell Medicine and the principal study investigator on the late-stage trial of Tavalisse, who has also served as a consultant and paid member of the advisory board for Rigel Pharmaceuticals. “The FDA approval of fostamatinib arms physicians with a new treatment option, which works via a novel mechanism.”

April 17, 2018
Photo: Raul Rodriguez, president and CEO of Rigel Pharmaceuticals.