FDA Accepts New Drug Application for Chronic ITP Treatment

"The U.S. Food and Drug Administration (FDA) filed Rigel Pharmaceuticals, Inc.’s New Drug Application for the use of Tavalisse (fostamatinib disodium) for the treatment of chronic immune thrombocytopenia (ITP).

Tavalisse, an oral investigational drug candidate designed to inhibit SYK kinase, a key enzyme in the immune process that leads to platelet destruction in ITP. It was previously granted Orphan Drug designation for treatment of the same condition in April.

ITP is a bleeding disorder in which blood doesn't clot as it does typically due to an unusually low number of platelets or thrombocytes. It’s almost always chronic in adults, but is often acute and self-limited in children. Symptoms include excessive bruising and bleeding.

'The FDA acceptance for filing of our NDA is an exciting milestone for Rigel," said Raul Rodriguez, Rigel's president and chief executive officer. "If approved, we believe Tavalisse will provide a new treatment option for patients with chronic or persistent ITP. We look forward to working closely with the FDA as they review our submission.'"

- By Mathew Shanley, Rare Disease Report

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