CAMBRIDGE, Mass., Aug. 7, 2019 /PRNewswire/ -- Blueprint Medicines
Corporation (NASDAQ: BPMC), a precision therapy company focused on
genomically defined cancers, rare diseases and cancer
immunotherapy, today announced that the U.S. Food and Drug
Administration (FDA) has accepted the company's New Drug
Application (NDA) for avapritinib for the treatment of adult
patients with PDGFRA Exon 18 mutant gastrointestinal stromal tumors
(GIST), regardless of prior therapy, and fourth-line GIST. The FDA
granted Priority Review and set an action date of February 14, 2020 under the Prescription Drug
User Fee Act (PDUFA). At this time, the FDA is not planning to hold
an advisory committee meeting to discuss this application.
Avapritinib, an investigational therapy, is a potent and highly
selective KIT and PDGFRA inhibitor for patients with advanced
GIST.
"Patients with PDGFRA Exon 18 mutant GIST and fourth-line GIST
are in need of new treatment options that address the underlying
drivers of the disease," said Andy
Boral, M.D., Ph.D., Chief Medical Officer at Blueprint
Medicines. "The FDA's acceptance of our application for Priority
Review brings us closer to our goal of delivering avapritinib to
patients with GIST, and we look forward to working closely with the
FDA during the review process."
The FDA's acceptance of the NDA indicates the application is
sufficiently complete to permit a substantive review. A Priority
Review designation accelerates the FDA's review time from 12 months
to a goal of eight months from the NDA submission date, and is
granted to drugs that may offer a significant improvement in the
safety or effectiveness of the treatment, prevention or diagnosis
of a serious condition. Previously, the FDA granted
avapritinib Breakthrough Therapy Designation for the treatment of
patients with unresectable or metastatic GIST harboring the PDGFRα
D842V mutation.
In July 2019, the European
Medicines Agency validated Blueprint Medicines' Marketing
Authorization Application for avapritinib in adult patients with
PDGFRα D842V mutant GIST, regardless of prior therapy, and
fourth-line GIST.
About GIST
GIST is a sarcoma, or tumor of bone or
connective tissue, of the gastrointestinal (GI) tract. Tumors arise
from cells in the wall of the GI tract and occur most often in the
stomach or small intestine. Most patients are diagnosed between the
ages of 50 to 80, and diagnosis is typically triggered by GI
bleeding, incidental findings during surgery or imaging and, in
rare cases, tumor rupture or GI obstruction.
Most GIST cases are caused by a spectrum of clinically relevant
mutations that force the KIT or PDGFRA protein kinases into an
increasingly active state. Because currently available therapies
primarily bind to the inactive protein conformations, certain
primary and secondary mutations typically lead to treatment
resistance and disease progression.
In unresectable or metastatic GIST, clinical benefits from
existing treatments can vary by mutation type. Mutational testing
is critical to tailor therapy to the underlying disease driver and
is recommended in expert guidelines. Currently, there are no
approved therapies for patients with KIT-driven GIST whose disease
progresses beyond imatinib, sunitinib and regorafenib. In patients
with metastatic PDGFRα D842V-driven GIST, progression occurs in a
median of approximately three to four months with available
therapy.
About Avapritinib
Avapritinib is an investigational,
oral precision therapy that selectively and potently inhibits KIT
and PDGFRA mutant kinases. It is a type 1 inhibitor designed to
target the active kinase conformation; all oncogenic kinases signal
via this conformation. Avapritinib has demonstrated broad
inhibition of KIT and PDGFRA mutations associated with GIST,
including potent activity against activation loop mutations that
are associated with resistance to currently approved therapies.
Blueprint Medicines is initially developing avapritinib for
the treatment of advanced GIST, advanced systemic mastocytosis
(SM), and indolent and smoldering SM. The FDA has granted
Breakthrough Therapy Designation to avapritinib for two
indications: one for the treatment of unresectable or metastatic
GIST harboring the PDGFRα D842V mutation and one for the treatment
of advanced SM, including the subtypes of aggressive SM, SM with an
associated hematologic neoplasm and mast cell leukemia.
Blueprint Medicines has an exclusive collaboration and license
agreement with CStone Pharmaceuticals for the development and
commercialization of avapritinib and certain other drug candidates
in Mainland China, Hong Kong,
Macau and Taiwan. Blueprint Medicines retains
development and commercial rights for avapritinib in the rest of
the world.
About Blueprint Medicines
Blueprint Medicines is
a precision therapy company striving to improve human health. With
a focus on genomically defined cancers, rare diseases and cancer
immunotherapy, we are developing transformational medicines rooted
in our leading expertise in protein kinases, which are proven
drivers of disease. Our uniquely targeted, scalable approach
empowers the rapid design and development of new treatments and
increases the likelihood of clinical success. We are currently
advancing four investigational medicines in clinical development,
along with multiple research programs. For more information,
visit www.BlueprintMedicines.com and follow us
on Twitter (@BlueprintMeds) and LinkedIn.
Cautionary Note Regarding Forward-Looking
Statements
This press release contains forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995, as amended, including, without limitation,
statements regarding the potential benefits of avapritinib in
treating patients with GIST; plans, timelines and expectations for
the FDA's review of the NDA for avapritinib for the treatment of
adult patients with PDGFRA Exon 18 mutant GIST, regardless of prior
therapy, and fourth-line GIST; plans, timelines and expectations
for the commercialization of avapritinib for the treatment of GIST,
if approved by the FDA; and Blueprint Medicines' strategy, goals
and anticipated milestones, business plans and focus. The words
"may," "will," "could," "would," "should," "expect," "plan,"
"anticipate," "intend," "believe," "estimate," "predict,"
"project," "potential," "continue," "target" and similar
expressions are intended to identify forward-looking statements,
although not all forward-looking statements contain these
identifying words. Any forward-looking statements in this press
release are based on management's current expectations and beliefs
and are subject to a number of risks, uncertainties and important
factors that may cause actual events or results to differ
materially from those expressed or implied by any forward-looking
statements contained in this press release, including, without
limitation, risks and uncertainties related to the delay of any
current or planned clinical trials or the development of Blueprint
Medicines' drug candidates, including avapritinib, pralsetinib,
fisogatinib and BLU-782; Blueprint Medicines' advancement of
multiple early-stage efforts; Blueprint Medicines' ability to
successfully demonstrate the safety and efficacy of its drug
candidates and gain approval of its drug candidates on a timely
basis, if at all; the preclinical and clinical results for
Blueprint Medicines' drug candidates, which may not support further
development of such drug candidates; actions of regulatory
agencies, which may affect the initiation, timing and progress of
clinical trials or the regulatory pathway; Blueprint Medicines'
ability to develop and commercialize companion diagnostic tests for
its current and future drug candidates; and the success of
Blueprint Medicines' current and future collaborations, including
its cancer immunotherapy collaboration with F. Hoffmann-La Roche
Ltd and Hoffmann-La Roche Inc. and its collaboration with CStone
Pharmaceuticals. These and other risks and uncertainties are
described in greater detail in the section entitled "Risk Factors"
in Blueprint Medicines' filings with the Securities and Exchange
Commission (SEC), including Blueprint Medicines' most recent
Quarterly Report on Form 10-Q and any other filings that Blueprint
Medicines has made or may make with the SEC in the future. Any
forward-looking statements contained in this press release
represent Blueprint Medicines' views only as of the date hereof and
should not be relied upon as representing its views as of any
subsequent date. Except as required by law, Blueprint Medicines
explicitly disclaims any obligation to update any forward-looking
statements.
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SOURCE Blueprint Medicines