TAVALISSE® (fostamatinib disodium hexahydrate) is a potent and selective oral spleen tyrosine kinase (SYK) inhibitor.
In May 2018, TAVALISSE was approved by the U.S. Food and Drug Administration (FDA) for use in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to at least one prior treatment. The patent for TAVALISSE expires in 2026, with an anticipated extension for patent restoration to 2032. We plan to take advantage of this long window of exclusivity and further explore its potential. Its ability to inhibit both Fc and B-cell receptor signaling pathways makes it a potentially broad immunomodulatory agent.
Currently, TAVALISSE is in late-stage clinical development in warm antibody autoimmune hemolytic anemia (AIHA).
Autoimmune Hemolytic Anemia (AIHA)
AIHA is a rare, serious blood disorder in which the immune system produces antibodies that result in the destruction (hemolysis) of the body’s own red blood cells (RBC). In AIHA, the body destroys RBCs at a greater rate than it can produce new RBCs, eventually leading to anemia (low levels of red blood cells), with symptoms such as fatigue, pale color, rapid heartbeat, and shortness of breath. In severe cases fever, chest pain, fainting, or heart failure may occur. Approximately 1 to 3 in 100,000 adults are diagnosed with AIHA each year. Warm antibody AIHA is the most common form of AIHA and can be either primary or secondary to an underlying disease such as systemic lupus erythematosus (SLE) or a lymphoproliferative condition such as or chronic lymphocytic leukemia (CLL) or lymphoma. To date, there are no approved therapies for AIHA, despite the unmet medical need.
Development of TAVALISSE in AIHA:
- In January 2018, FDA granted Orphan Drug designation for the treatment of patients with warm antibody AIHA
- Topline results of Phase 2 clinical trial (SOAR)
- Ongoing Phase 3 clinical trial (FORWARD) began enrolling patients in May 2019
- Approximately 80 patients in a 24-week study
- Topline results projected for mid-2021