U.S. FDA approves Blueprint’s therapy for rare type of stomach cancer

(Reuters) – The U.S. Food and Drug Administration approved Blueprint Medicines Corp’s drug to treat a rare form of cancer that affects the stomach and small intestine, the agency said on Thursday.

FILE PHOTO: A view shows the U.S. Food and Drug Administration (FDA) headquarters in Silver Spring, Maryland August 14, 2012. REUTERS/Jason Reed/File Photo

The agency’s decision makes Blueprint’s oral drug, Ayvakit, the first approved treatment for a small subset of patients with a mutation of the cancer called gastrointestinal stromal tumor (GIST), according to the company.

GIST tumors, which usually begin in the stomach or small intestine, are a type of soft-tissue sarcoma. Patients are at risk of the cancer recurring even after successful surgery to remove tumors.

About 5,000 Americans are diagnosed with the cancer every year. With Ayvakit, Blueprint is looking to target about 6% of these patients whose cancers are driven by PDGFRA exon 18 mutations.

In patients with the mutation, the cancer tends to progress in a median of about three to four months after being treated with currently available therapies.

Ayvakit works by selectively inhibiting the drivers of the mutation and was found to be well tolerated and effective in 86% of patients it was tested in, the company said.

“We’re not only having significant impact on shrinking their tumors, we’re also keeping them disease progression free for very long periods of time,” Chief Operating Officer Kate Haviland told Reuters in a phone interview.

Balancing the off-target effects of the therapy has also helped patients stay on the treatment, said Haviland, adding that the company has seen patients continue the therapy for nearly four years.

GIST patients who have undergone surgery are typically given Novartis AG’s Gleevec for 12 months in an effort to delay the spread or recurrence of cancer. However, the drug has not been effective in treating patients with the mutation targeted by Blueprint.

Blueprint is also pursuing FDA approval for use of Ayvakit in patients in advanced stages of GIST who have exhausted all other options of care – a much larger population of patients. The company is expected to announce top-line data from its trials in the second quarter of 2020.

Reporting by Vishwadha Chander and Saumya Sibi Joseph in Bengaluru; Editing by Aditya Soni

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