The US Food and Drug Administration (FDA) has just approved a new drug called Tavalisse™ to treat adults who have thrombocytopenia as a result of chronic immune thrombocytopenia (ITP), following the failure of a previous treatment.

To read the full press release including a video from the developers, Rigel Pharmaceuticals, click here.

ITP is a rare autoimmune bleeding disorder that prevents blood from clotting normally. The immune systems of people with ITP mistakenly attack the body’s own platelet cells. Platelets, or thrombocytes, are found in the blood and one of their roles is to stick together to form blood clots. The low platelet counts of people with ITP prevent this from occurring, and as a result bleeding often continues for a long time. This causes many of the symptoms seen in ITP patients including bruises that can be seen on the skin or mucous membranes (e.g. inside the mouth), and rash-like small red dots under the skin caused by internal bleeding. Patients may also experience nosebleeds, gum bleeding, heavy menstrual bleeding, and hematomas (lumps under the skin caused by clotted blood).

Existing treatment options for those with ITP include medicines such as steroids, and surgery to remove a patient’s spleen, which produces the antibodies that destroy the platelets. However, these treatments are not effective for all patients. The recently approved drug, Tavalisse, uses a new mechanism to fight the disease. The drug inhibits a signaling protein in the spleen called spleen tyrosine kinase (SYK) that is involved in the process of platelet destruction in ITP patients. Inhibition of SYK therefore reduces platelet destruction causing an improvement in patient symptoms. Tavalisse was approved by the FDA following a Phase 3 clinical trial that showed the drug was effective compared to a placebo, and data from 163 patients with ITP who trialled it.

Tavalisse will provide patients and doctors with another treatment option for chronic immune thrombocytopenia. More information about the drug can be found at www.tavalisse.com, and it is planned to become available in the US in late May 2018.

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