FDA approves new AML drug after clinical testing at Dana-Farber

May 5 2017

A targeted drug whose clinical testing was led by Richard Stone, MD, of Dana-Farber Cancer Institute, has become the first new treatment for newly diagnosed acute myeloid leukemia (AML) in more than 25 years.

The drug, midostaurin (Rydapt^®), was approved by the U.S. Food and Drug Administration (FDA) as a combination treatment, with chemotherapy, for adult patients newly diagnosed with AML that carries a mutation in the gene FLT3. Such patients account for roughly a third of the 21,000 Americans diagnosed annually with AML, a rare and aggressive disease of the blood and bone marrow.

"The overall survival advantage for midostaurin plus chemotherapy seen in the RATIFY trial was a significant advancement for newly diagnosed AML patients with the FLT3 mutation," Stone said. "The availability of midostaurin now helps to establish a new standard of care in this high-risk patient population."

The benefits of midostaurin for this group of patients were borne out in the RATIFY trial, an international phase 3 clinical trial led by Stone, chief of staff and director of the Adult Leukemia Program at Dana-Farber. The trial, which included 717 patients with previously untreated FLT3-mutated AML, found that those treated with midostaurin and chemotherapy lived significantly longer and had a 23 percent lower risk of death than those treated with chemotherapy alone.

FLT3 is a cell surface protein cells that plays a role in increasing the number of certain blood cells. The FLT3 gene mutation can result in faster progression of the disease, higher relapse rates, and lower survival rates than other forms of AML. Prior to the approval of midostaurin, the treatment of AML had been relatively unchanged for more than 25 years.

In the trial, the most frequent adverse side effects in patients receiving midostaurin plus chemotherapy included low white blood cell counts, nausea, pain or ulcers inside the mouth, vomiting, headache, bruising, musculoskeletal pain, nose bleeds, device-related infections, high blood sugar levels, and upper respiratory tract infections.