Tavalisse

Generic name: fotantinib

Trade name: Tavalisse

Full names: fotantinib, Tavalisse, fostamatinib

Indications:

Treatment of adult patients with chronic immune thrombocytopenia (ITP).

The results of the phase 3 clinical study (n=34) showed that the proportion of patients in the Tavalisse treatment group achieving stable platelet response was significantly higher than that in the placebo group. A stable platelet response was defined as a blood platelet count of ≥50,000 platelets/μL on at least 4 of the last 6 planned visits during weeks 14-24 of treatment. In this study, the safety profile of Tavalisse was consistent with other clinical trials, and no new or unusual safety issues were identified.

In October 2018, Rigel signed an exclusive license and supply agreement with Kissei to develop all existing and potential indications of fostamatinib in Japan, China, and South Korea. In the United States, fostamatinib was approved in April 2018, marketed under the trade name Tavalisse, for the treatment of adult patients with chronic ITP who have had an inadequate response to prior therapy.

Immune thrombocytopenia (ITP) is a rare, serious autoimmune disease characterized by the immune system attacking and destroying its own platelets, resulting in low platelet counts in the blood (thrombocytopenia), easy bleeding, and adverse effects on the patient's quality of life. Patients with chronic ITP are at increased risk for serious bleeding events, leading to serious medical complications and even death. Current treatments for ITP include steroids, blood cell production-enhancing agents (TPO receptor agonists), and splenectomy. However, not all patients can be adequately treated with existing therapies. Therefore, ITP patients still need more treatment options.

Tavalisse is an oral splenic tyrosine kinase (SYK) inhibitor that addresses the underlying autoimmune cause of the disease by blocking platelet destruction, providing an important new treatment option for adults with chronic ITP.

Rigel Executive Vice President and Chief Medical Officer Wolfgang "These Phase 3 study results add to the growing body of evidence that fostamatinib may provide meaningful benefits to patients with ITP," said Dr. Dummer. "Fostamatinib has a unique mechanism of action that addresses the underlying autoimmune cause of ITP by blocking platelet destruction. We are also continuing to evaluate fostamatinib's potential to treat warm antibody autoimmune hemolytic anemia (waAIHA), another rare blood disorder in which the immune system destroys blood cells."