Blueprint Medicines won approval Thursday of its first drug, a precision medicine that dramatically shrank tumors caused by an ultra-rare form of gastrointestinal cancer.
The approval by the Food and Drug Administration of the medicine for a form of gastrointestinal stromal tumors, or GIST, marks an “incredibly exciting milestone for our company and, more importantly, for GIST patients,” said Jeff Albers, chief executive of the Cambridge biotech.
The list price of the drug, which is called Ayvakit, is $32,000 for a 30-day supply.
The FDA action was widely expected, and Blueprint shares on the Nasdaq closed up less than 1 percent.
Ayvakit is the first effective treatment for GIST patients with a particular mutation, according to Dr. Suzanne George, an oncologist at the Dana-Farber Cancer Institute. Those patients are believed to number in the hundreds in the United States.
“It’s going to change the standard of care for this particular subset of patients with GIST,” said George, principal investigator at Dana-Farber, one of 19 sites in the United States and overseas where the drug was tested. “If you ask me, it already has.”
Five GIST patients with the rare mutation were treated with Ayvakit in the clinical trial at Dana-Farber. A total of 56 patients with the mutation received the drug at all the sites.
“The response rate is occurring in almost all the patients treated,” George said.
Before Blueprint began testing the experimental medicine, she added, patients with the rare form of gastrointestinal stromal tumors typically underwent multiple surgeries or were treated with other cancer drugs, such as Gleevec, a kinase inhibitor, which were less effective..
Among the patients who participated in the trial at Dana-Farber was Mindy Schutte, a 38-year-old mother of three who lives in a wooded area in northern Wisconsin, near Lake Superior.
Schutte said her abdomen didn’t shrink after she delivered her third child in 2011. After imaging scans detected a mass, she underwent surgery in 2012 at the hospital at the University of Minnesota and the surgeon removed a 2-pound gastrointestinal stromal tumor dangling from her stomach.
She initially took Gleevec to prevent the cancer from growing back. But in 2016 she was scanned again and doctors found at least six new tumors on her pelvis, uterus, and right lung. She underwent surgery again, she said, but the tumors grew back despite her taking Gleevec.
In 2017, Schutte began taking Blueprint’s experimental drug at Dana-Farber. It initially caused her hair to turn white and made her eyes look jaundiced, she said. Doctors lowered the dosage, and the tumors shrank. She had a CT scan last August, and doctors were unable to detect the cancer.
“This is the first thing that worked without me having to be cut wide open,” she said. “I was out of options.”
Dr. Michael Heinrich, a professor of medicine at the Oregon Health & Science University and also an investigator in the clinical trial, said the FDA approval should prompt doctors to do genetic testing of GIST patients to determine if they might benefit from the new drug.
“Building on our growing understanding of the molecular basis of GIST, this milestone ushers in a new era of precision medicine in this disease,” he said.
Blueprint was founded in 2008 with financing led by Third Rock Ventures. The company has about 380 employees.
Jonathan Saltzman can be reached at firstname.lastname@example.org.