Rigel Pharmaceuticals has bagged its first FDA approval, with a green light to market Tavalisse for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have failed to respond to previous treatment.
Chronic ITP is a rare, autoimmune disease in which the immune system destroys platelets, which are necessary for normal blood clotting. The disorder is difficult to treat because it is impossible to predict how people will respond to available therapies.
Around 50,000-60,000 adults live in the with primary ITP, and many do not respond or stop responding to one or more current therapies, the firm noted, highlighting the unmet need in the area.
Tavalisse (fostamatinib disodium hexahydrate), an SYK inhibitor, has a unique mechanism of action in that it targets the underlying autoimmune cause of the disease by impeding platelet destruction.
"We are excited to bring this new medicine to the population of adult patients with chronic ITP in need of additional therapies,” said Raul Rodriguez, president and chief executive of Rigel.
"This regulatory milestone, our first product approval, validates the therapeutic effect of SYK inhibition in an autoimmune disease."
Rigel says it plans to launch the drug in the US next month.