Investigational drug for GIST is on the fast track

The Food and Drug Administration has decided to grant regorafenib fast-track status. The drug is in phase 3 testing for the treatment of gastrointestinal stromal tumors (GIST), a rare type of sarcoma. Fast tracking typically helps move drugs that fill unmet needs and treat serious conditions, such as GIST, as fast as possible through the pipeline. The phase 3 clinical trial, which started in January, will be examining 170 patients with inoperable GIST who will receive either regorafenib or placebo. (If patients who are on placebo have disease progression, they may be allowed to receive regorafenib.) Data from a phase 2 trial of regorafenib will be presented next month at the annual meeting of the American Society of Clinical Oncology. Other drugs approved for GIST include Gleevec (imatinib) and Sutent (sunitinib). Patients in the phase 3 trial will have progressed on either drug. Unlike Gleevec, which targets one specific mutation called bcr-abl, regorafenib is a multikinase inhibitor, acting against several signals, including VEGF, RAF and KIT. For more on fast-track status and other drug approval processes, see "A Primer on How Faster Approval Works." For more information on GIST, the organizations listed below have resource, educational materials and support services: GIST Support International

gistsupport.org

215-340-9374

E-mail: gsi@gistsupport.org

This organization is dedicated to the support of GIST patients, families and friends, and the ongoing research required to treat and cure gastrointestinal stromal tumors. Life Raft Group

liferaftgroup.org

973-837-9092

The Life Raft Group is a non-profit, Internet-based organization providing support through education and research to patients with gastrointestinal stromal tumors. For pediatric GIST patients, the Life Raft Group also hosts pediatricgist.org, which offers information, newsletters, community and resources.