CAMBRIDGE, Mass., May 15, 2020 /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 issued a complete response letter (CRL)
for the new drug application of avapritinib for the treatment of
adults with unresectable or metastatic fourth-line gastrointestinal
stromal tumor (GIST). The CRL states that the FDA cannot approve
the application.
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As previously announced, Blueprint Medicines plans to continue
to commercialize AYVAKIT™ (avapritinib) in the United States for the treatment of adults
with unresectable or metastatic GIST harboring a PDGFRA exon 18
mutation, including PDGFRA D842V mutations, and seek marketing
approval for avapritinib for the treatment of this patient
population in additional geographies, including the European Union.
In addition, Blueprint Medicines continues to advance development
of avapritinib for the treatment of systemic mastocytosis (SM).
Based on top-line results reported in April
2020 for Blueprint Medicines' Phase 3 VOYAGER trial, the
company previously announced plans to discontinue further
development of avapritinib in GIST indications other than PDGFRA
exon 18 mutant GIST.
About AYVAKIT (avapritinib)
AYVAKIT (avapritinib) is a kinase inhibitor approved by the FDA
for the treatment of adults with unresectable or metastatic GIST
harboring a PDGFRA exon 18 mutation, including PDGFRA D842V
mutations. AYVAKIT is the first precision therapy approved to treat
a genomically defined population of patients with GIST and the only
highly active treatment for PDGFRA exon 18 mutant GIST. The FDA
granted Breakthrough Therapy Designation to avapritinib for the
treatment of unresectable or metastatic GIST harboring the PDGFRA
D842V mutation. For more information, visit AYVAKIT.com.
Avapritinib is not approved for the treatment of any other
indication in the U.S. or any other jurisdiction by the FDA or any
other health authority.
Blueprint Medicines is developing avapritinib globally for
the treatment of advanced, smoldering and indolent SM. The FDA
granted Breakthrough Therapy Designation to avapritinib 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 have one FDA-approved precision therapy and
are currently advancing multiple investigational medicines in
clinical development, along with a number of 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 plans to continue to commercialize AYVAKIT in
the United States for the
treatment of adults with unresectable or metastatic GIST harboring
a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and
plans to seek marketing approval for avapritinib for the treatment
of this patient population in additional geographies; plans to
discontinue further development of avapritinib for GIST indications
other than PDGFRA exon 18 mutant GIST; the potential benefits of
Blueprint Medicines' current and future drug candidates in treating
patients; and Blueprint Medicines' strategy, goals and anticipated
milestones, business plans and focus. The words "aim," "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 impact of the COVID-19 pandemic to
Blueprint Medicines' business, operations, strategy, goals and
anticipated milestones, including Blueprint Medicines' ongoing and
planned research and discovery activities, ability to conduct
ongoing and planned clinical trials, clinical supply of current or
future drug candidates, commercial supply of current or future
approved products, and the launch, marketing and sale of current or
future approved products; Blueprint Medicines' ability and plan in
establishing a commercial infrastructure, and successfully
launching, marketing and selling its approved product; Blueprint
Medicines' ability to successfully expand the approved indications
for AYVAKIT or obtain marketing approval for AYVAKIT in additional
geographies in the future; the delay of any current or planned
clinical trials or the development of Blueprint Medicines' drug
candidates or licensed product candidate; Blueprint Medicines'
advancement of multiple early-stage efforts; Blueprint Medicines'
ability to successfully demonstrate the efficacy and safety 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; 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 or licensing arrangements. 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 Annual Report on Form 10-K, as
supplemented by its 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 Corporation