CAMBRIDGE, Mass., Feb. 6, 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 extended the Prescription Drug User Fee
Act (PDUFA) date for its New Drug Application (NDA) seeking
accelerated approval of avapritinib for the treatment of adults
with fourth-line gastrointestinal stromal tumor (GIST). The FDA
extended the PDUFA action date by three months from February 14, 2020 to May
14, 2020.
As previously announced, the FDA has requested top-line data
from Blueprint Medicines' ongoing Phase 3 VOYAGER clinical trial,
which is comparing avapritinib to regorafenib in third- or
fourth-line GIST, to inform the pending action on the NDA for
fourth-line GIST. Blueprint Medicines expects to provide the
top-line data to the FDA early in the second quarter of 2020 to
enable the FDA to take action by the May 14,
2020 PDUFA date.
In addition, Blueprint Medicines today announced that the
National Comprehensive Cancer Network has updated its Clinical
Practice Guidelines in Oncology (NCCN Guidelines®) for
Soft Tissue Sarcoma to include avapritinib as a recommended
category 2A treatment for unresectable or metastatic GIST with a
PDGFRA exon 18 mutation, including PDGFRA D842V mutations, as well
as fourth-line GIST. This rating indicates that there is uniform
NCCN consensus that the intervention is appropriate. The NCCN
Guidelines are the recognized clinical standard for cancer care by
U.S. healthcare providers and payers, and are maintained by a
committee of expert physicians from leading U.S. cancer
centers.
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 a selective and potent inhibitor of KIT and
PDGFRA mutant kinases. It is the only FDA-approved type 1 inhibitor
for GIST that works by directly binding to the active kinase
conformation from which mutant KIT and PDGFRA signal. AYVAKIT has
demonstrated inhibition of a broad range of KIT and PDGFRA
mutations associated with GIST, including potent clinical activity
against activation loop mutations that are associated with
resistance to currently approved therapies. For more information,
visit AYVAKIT.com.
Avapritinib is not approved for the treatment of any other
indication, including fourth-line GIST, in the U.S. or any other
jurisdiction by the FDA or any other health authority.
Blueprint Medicines is pursuing a broad clinical development
program for avapritinib across multiple lines of GIST treatment, as
well as for advanced, smoldering and indolent systemic mastocytosis
(SM). The FDA has granted Breakthrough Therapy Designation to
avapritinib for two indications: one for the treatment of
unresectable or metastatic GIST harboring the PDGFRA 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. For more information about avapritinib clinical
trials, visit www.clinicaltrials.gov or
www.blueprintclinicaltrials.com.
Important Safety Information
Intracranial hemorrhage (e.g., subdural hematoma, intracranial
hemorrhage, and cerebral hemorrhage) occurred in 1% of 267 patients
(0.7% Grade 3 or 4) with GIST and overall in 3% of 335 patients
(1.2% Grade 3 or 4) who received AYVAKIT. Overall, 0.9% of patients
receiving AYVAKIT required permanent discontinuation for an
intracranial hemorrhage. Withhold AYVAKIT and then resume at a
reduced dose upon resolution, or permanently discontinue AYVAKIT
based on severity.
In 335 patients receiving AYVAKIT, CNS adverse reactions
occurred overall in 58% of patients including cognitive impairment
(41%; 3.6% Grade 3 or 4), dizziness (20%; 0.6% Grade 3 or 4), sleep
disorders (15%; 0.3% Grade 3 or 4), mood disorders (13%; 1.5% Grade
3 or 4), speech disorders (6%; none Grade 3 or 4), and
hallucinations (2.1%; none Grade 3 or 4). Overall, 3.9% of patients
required permanent discontinuation of AYVAKIT for a CNS adverse
reaction. Depending on severity, withhold AYVAKIT and then resume
at the same dose or at a reduced dose upon improvement, or
permanently discontinue AYVAKIT.
AYVAKIT can cause fetal harm when administered to a pregnant
woman. Advise females of reproductive potential and pregnant women
of the potential risk to a fetus. Advise females and males of
reproductive potential to use an effective method of contraception
during treatment with AYVAKIT and for 6 weeks after the final dose
of AYVAKIT. Advise women not to breastfeed during treatment with
AYVAKIT and for two weeks after the final dose. Advise females and
males of reproductive potential that AYVAKIT may impair
fertility.
In 204 patients with unresectable or metastatic GIST, the most
common adverse reactions (≥ 20%) were edema, nausea,
fatigue/asthenia, cognitive impairment, vomiting, decreased
appetite, diarrhea, hair color changes, increased lacrimation,
abdominal pain, constipation, rash and dizziness.
Avoid coadministration of AYVAKIT with strong and moderate CYP3A
inhibitors. If coadministration with a moderate CYP3A inhibitor
cannot be avoided, reduce dose of AYVAKIT. Avoid coadministration
of AYVAKIT with strong and moderate CYP3A inducers.
Please click here to see the full Prescribing
Information for AYVAKIT.
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 the potential benefits of avapritinib in treating
patients with GIST; plans, timelines and expectations for top-line
data from the VOYAGER trial; plans to prioritize completion of the
VOYAGER trial; timelines and expectations regarding an FDA decision
on the NDA for fourth-line GIST; 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; 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; 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, including its
cancer immunotherapy collaboration with F. Hoffmann-La Roche Ltd
and Hoffmann-La Roche Inc., its collaboration with CStone
Pharmaceuticals and its license to Clementia 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 Corporation