Blueprint Medicines Corp.'s targeted cancer drug Ayvakit has been approved by the U.S. Food and Drug Administration for treating certain gastrointestinal tumors that can not be surgically removed or have spread to other body parts.
Ayvakit is the first drug approved to treat patients with gastrointestinal stromal tumor, or GIST, who have a specific mutation of the platelet-derived growth factor receptor alpha, or PDGFRA gene, the U.S. regulator noted in a Jan. 9 release. Tumors with this mutation do not respond to the usual course of treatment.
GIST is a type of sarcoma or cancer that develops in the bone or connective tissue. These tumors grow in the wall of the gastrointestinal tract, most commonly in the stomach or small intestine. Most cases of GIST are caused by genetic mutations that force proteins into an active state.
The approval is based on results from a phase 1 clinical trial called Navigator, which showed a reduction of tumor size in 84% of patients with the PDGFR mutation.
Ayvakit, or avapritinib, blocks a particular enzyme and prevents the cancer cells from growing.
Cambridge, Mass.-based Blueprint Medicines plans to launch the drug in the U.S. market within a week, according to a press release. According to one doctor involved in the Navigator trial, the FDA's nod "represents a call to action" for GIST patients to be tested for this gene mutation to apply the precision medicine approach before other treatment is offered.
Blueprint Medicines' application for Ayvakit was broken down by the FDA into two parts in October 2019. The regulator expects to announce a decision regarding the second indication of the drug as a fourth-in-line treatment for GIST by Feb. 14.
The FDA has also granted Ayvakit breakthrough therapy designation for treating advanced systemic mastocytosis, a disorder in which mast cells accumulate in internal tissues and organs such as the liver, spleen, bone marrow and small intestines.