-- FDA grants priority review and sets PDUFA
action date of May 22, 2023
--
CAMBRIDGE, Mass., Jan. 23,
2023 /PRNewswire/ -- Blueprint Medicines Corporation
(NASDAQ: BPMC) today announced that the U.S. Food and Drug
Administration (FDA) has accepted the company's supplemental new
drug application for AYVAKIT® (avapritinib) for the
treatment of adults with indolent systemic mastocytosis (SM). The
FDA granted priority review with an action date of May 22, 2023 under the Prescription Drug User Fee
Act (PDUFA).
This regulatory application is based on results from the global
PIONEER trial, the largest randomized, placebo-controlled study
ever conducted in indolent SM. The FDA previously granted
breakthrough therapy designation to AYVAKIT for the treatment of
moderate to severe indolent SM. AYVAKIT was designed to potently
and selectively inhibit D816V mutant KIT, the primary underlying
cause of SM.
"People with indolent systemic mastocytosis experience
debilitating symptoms and poor quality of life, and we have the
potential to transform clinical outcomes for these patients by
targeting the genetic driver of disease with AYVAKIT," said Becker
Hewes, M.D., Chief Medical Officer at Blueprint Medicines.
"AYVAKIT achieved the primary and all key secondary endpoints in
the PIONEER trial, with highly meaningful reductions in
patient-reported symptoms and all measures of mast cell burden
studied, and a well-tolerated safety profile supporting chronic
treatment. We look forward to collaborating with the FDA during its
review process, with the goal of bringing the first approved
medicine to patients with indolent SM and redefining the treatment
landscape beyond symptom-directed therapies."
About AYVAKIT (avapritinib)
AYVAKIT (avapritinib) is a kinase inhibitor approved by the FDA
for the treatment of adults with Advanced SM, including aggressive
SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and
mast cell leukemia (MCL), and adults with unresectable or
metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA
exon 18 mutation, including PDGFRA D842V mutations. For more
information, visit AYVAKIT.com. This medicine is approved
in Europe (AYVAKYT®) for the treatment of
adults with ASM, SM-AHN or MCL, after at least one systemic
therapy, and adults with unresectable or metastatic GIST harboring
the PDGFRA D842V mutation. Please click here to see the
full U.S. Prescribing Information for AYVAKIT, and
click here to see the European Summary of Product
Characteristics for AYVAKYT. AYVAKIT/AYVAKYT is not approved
for the treatment of any other indication in
the U.S. or Europe.
To learn about ongoing or planned clinical trials,
contact Blueprint Medicines at
medinfo@blueprintmedicines.com or 1-888-BLU-PRNT
(1-888-258-7768). Additional information is available
at blueprintclinicaltrials.com or clinicaltrials.gov.
About Systemic Mastocytosis
Systemic mastocytosis (SM) is a rare disease driven by the KIT
D816V mutation in about 95 percent of cases. Uncontrolled
proliferation and activation of mast cells result in chronic,
severe and often unpredictable symptoms across multiple organ
systems. Most of those affected have non-advanced (indolent or
smoldering) SM, and among these patients, the vast majority have
indolent SM. A broad range of symptoms, including anaphylaxis,
maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone
pain, frequently persist in patients with non-advanced SM despite
treatment with multiple symptom-directed therapies. This burden of
disease can lead to a profound, negative impact on quality of life.
Patients often live in fear of severe, unexpected symptoms, have
limited ability to work or perform daily activities, and isolate
themselves to protect against unpredictable triggers. Currently,
there are no approved therapies for the treatment of non-advanced
SM.
A minority of patients have advanced SM, which encompasses a
group of high-risk SM subtypes including ASM, SM-AHN and MCL. In
addition to mast cell activation symptoms, advanced SM is
associated with organ damage due to mast cell infiltration and poor
survival.
About Blueprint Medicines
Blueprint Medicines is a global precision therapy company
that invents life-changing therapies for people with cancer and
blood disorders. Applying an approach that is both precise and
agile, we create medicines that selectively target genetic drivers,
with the goal of staying one step ahead across stages of disease.
Since 2011, we have leveraged our research platform, including
expertise in molecular targeting and world-class drug design
capabilities, to rapidly and reproducibly translate science into a
broad pipeline of precision therapies. Today, we are delivering our
approved medicines to patients in the United States and Europe, and we are
globally advancing multiple programs for systemic mastocytosis,
lung cancer, breast cancer and other genomically defined cancers,
and cancer immunotherapy. For more information,
visit www.BlueprintMedicines.com and follow us on
Twitter (@BlueprintMeds) and LinkedIn.
Cautionary Note Regarding Forward-Looking Statements
This presentation contains forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995, as
amended, including, without limitation, statements regarding plans,
strategies, timelines and expectations for interactions with the
U.S. Food and Drug Administration (FDA) and other regulatory
authorities; statements regarding the plans and potential benefits
of AYVAKIT in treating patients with indolent SM; statements
regarding plans and expectations for Blueprint Medicines' current
or future approved drugs and drug candidates; the potential
benefits of any of the company's current or future approved drugs
or drug candidates in treating patients; and Blueprint Medicines'
financial performance, 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 launching, marketing and selling current or
future approved products; Blueprint Medicines' ability and plans in
continuing to establish and expand a commercial infrastructure, and
successfully launching, marketing and selling current or future
approved products; Blueprint Medicines' ability to successfully
expand the approved indications for AYVAKIT/AYVAKYT or obtain
marketing approval for AYVAKIT/AYVAKYT in additional geographies in
the future; the delay of any current or planned clinical trials or
the development of Blueprint Medicines' current or future drug
candidates; 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 either as monotherapies or in combination with other
agents or may impact the anticipated timing of data or regulatory
submissions; the timing of the initiation of clinical trials and
trial cohorts at clinical trial sites and patient enrollment rates;
actions of regulatory agencies, which may affect the initiation,
timing and progress of clinical trials; Blueprint Medicines'
ability to obtain, maintain and enforce patent and other
intellectual property protection for AYVAKIT/AYVAKYT or any drug
candidates it is developing; Blueprint Medicines' ability to
develop and commercialize companion diagnostic tests for
AYVAKIT/AYVAKYT or any of its current and future drug candidates;
Blueprint Medicines' ability to successfully expand its operations,
research platform and portfolio of therapeutic candidates, and the
timing and costs thereof; and the success of Blueprint Medicines'
current and future collaborations, financing arrangements,
partnerships 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.
Trademarks
Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are
trademarks of Blueprint Medicines Corporation.
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SOURCE Blueprint Medicines Corporation