Rigel Pharmaceuticals’ ITP therapy Tavalisse (fostamatinib) is now available for Expanded Access/ Compassionate Use. Fostamatinib works by inhibiting platelet destruction in antibody-mediated platelet elimination by blocking a key part of the signaling mechanism in this critical pathway. The therapy may be a good option for patients who have been unresponsive to other past ITP treatments due to its different mechanism of action. “Compassionate Use” is granted by the FDA for therapies still under clinical trial review, to patients living with rare diseases for which there are no comparable alternatives for treatment. While clinical trials for new drugs are used to compile data on the safety and efficacy of a new drug, compassionate use programs are used solely to treat patients and do not gather information about the therapy. Physicians can request compassionate use of fostamatinib if the patient is not eligible to enroll in Rigel’s clinical trial and no alternative treatment exists. Treating physicians can make a request via completion of an application at
eap.rigel.com
. New requests will be considered until April 28 2018.
pdsa.org/products-a-publications/e-news/2018-enews/item/1617-pdsa-enews-042018.html?bblinkid=91950053&bbemailid=7574052&bbejrid=600117199#compassionate-use
