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 The FDA's decision makes the therapy, Ayvakit, the first treatment
for a small subset of patients with a mutation of the cancer called
gastrointestinal stromal tumor (GIST).
Blueprint said it expects to make Ayvakit available through a
limited specialty pharmacy network in the United States within a
week, with a wholesale acquisition cost or list price of $32,000 for
a 30-day supply, regardless of dosage.
The list price is not necessarily what patients actually pay as
"out-of-pocket" costs vary based on the duration of the treatment
and individual healthcare plans.
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 more than 80% of
patients it was tested in, the company said.
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"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 off-target effects has 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. However, the drug has not been effective in
treating patients with the mutation targeted by Blueprint.
"It's a big improvement for patients and a very big milestone for
the company... but in terms of sales, this exact indication is not
huge," said Cowen and Co analyst Marc Frahm who expects Ayvakit peak
sales of $68 million by 2032.
Blueprint is also pursuing FDA approval for Ayvakit in patients with
GIST who have exhausted all other options of care - a much larger
population of patients.
(Reporting by Vishwadha Chander and Saumya Sibi Joseph in Bengaluru;
Editing by Aditya Soni)
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