Oral SYK Inhibitor for ITP:
- Immune Thrombocytopenic Purpura (ITP) is an autoimmune disease where the immune system attacks and destroys platelets in the blood. The result - abnormally low platelet counts.
- There are two forms of ITP: acute thrombocytopenic purpura, which is most commonly seen in young children; and chronic thrombocytopenic purpura, which requires continual follow up care with a hematologist.
- There are approximately 60,000 to 125,000 patients with ITP in the U.S., and it qualifies as an orphan disease.
- Rigel is focusing its Fostamatinib ITP program on the chronic form of this disorder targeting the underlying autoimmune cause of the disease rather than stimulating platelet production.
- Antibodies, usually of the IgG type, mediate platelet destruction in ITP. Fostamatinib has a novel mechanism of action, blocking IgG receptor signaling in both macrophages and B cells via SYK kinase.
- Rigel completed and reported results from the FIT Phase 3 clinical program of fostamatinib in ITP in 2016.
- Rigel submitted an NDA to the FDA for fostamatinib in patients with chronic or persistent ITP in April 2017.