In April of this year, Rigel Pharmaceuticals, Inc. announced that it has submitted a New Drug Application (NDA) to the U.S. FDA for fostamatinib, a drug for patients with chronic idiopathic thrombocytopenic purpura (ITP).
This application is supported by data from a Phase 3 clinical trial in 2016 consisting of three studies, two of which were randomized placebo-controlled studies. In total, fostamatinib was evaluated in over 4,600 subjects.
Since this submission, fostamatinib has been proprietarily named Tavalisse™.
Even though the drug is not yet officially approved, Raul Rodriguez, the president and CEO of Rigel, said that the NDA submission of Tavalisse™ in and of itself is
“a major milestone in bringing new treatment options to patients suffering from this disease.”
This application is hugely significant for the ITP community because current therapies like steroids, blood platelet production boosters, and splenectomies do not always benefit every patient. Tavalisse™, on the other hand, is an oral investigational drug that is able to inhibit SYK (spleen tyrosine kinase), which leads to the platelet destruction characteristic of ITP.
In this way, Tavalisse™ addresses the underlying autoimmune cause of ITP, instead of merely controlling the problems of the immune system.
Notification of the results of the New Drug Application are expected in summer of 2017, and the Orphan Drug designation for Tavalisse™ has already been granted.
Stay tuned and keep Tavalisse™ on your radar because big things are motion for this exciting new drug!
In the meantime, if you want to look at the clinical trial reports for fostamatinib, click here!