-- Company to present registrational PIONEER
trial results at 2023 AAAAI Annual Meeting --
CAMBRIDGE, Mass., Nov. 22,
2022 /PRNewswire/ -- Blueprint Medicines
Corporation (NASDAQ: BPMC) today announced the submission
of a supplemental new drug application to the U.S. Food and
Drug Administration (FDA) for AYVAKIT® (avapritinib) for the
treatment of adults with indolent systemic mastocytosis (SM).
AYVAKIT was designed to potently and selectively inhibit D816V
mutant KIT, the underlying cause of SM in about 95 percent of
cases.
The submission includes data from the registrational PIONEER
trial, the largest randomized, placebo-controlled clinical study
ever conducted in indolent SM. The FDA has granted breakthrough
therapy designation to AYVAKIT for the treatment of moderate to
severe indolent SM.
"This regulatory submission for AYVAKIT marks an important
milestone to address the medical needs of a broad population of
patients with systemic mastocytosis, characterized by debilitating
symptoms and lifelong disease burden across multiple organs," said
Becker Hewes, M.D., Chief Medical Officer at Blueprint
Medicines. "By targeting the primary driver of SM with AYVAKIT, we
are seeking to transform treatment beyond symptom-directed therapy,
modify the course of disease and ultimately, make a meaningful
difference in patients' lives. Our FDA filing marks the first
regulatory application seeking approval for the treatment of
indolent SM, and we look forward to working closely with the agency
toward our goal of bringing AYVAKIT to indolent SM patients as
quickly as possible."
In addition, Blueprint Medicines today announced the acceptance
of multiple abstracts highlighting registrational PIONEER trial
data for oral and poster presentation at the American Academy of
Allergy, Asthma & Immunology (AAAAI) Annual Meeting,
February 24-27, 2023. AAAAI plans to
post accepted abstracts at www.annualmeeting.aaaai.org on
February 3, 2023.
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 primarily driven by
the KIT D816V mutation. Uncontrolled proliferation and activation
of mast cells result in chronic, severe and often unpredictable
symptoms across multiple organ systems. The vast majority of those
affected have non-advanced (indolent or smoldering) 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 our scientific
innovation into a broad pipeline of important approved and
investigational precision therapies aimed at addressing
difficult-to-treat cancers and blood disorders. Today, we are
delivering our approved medicines to patients in the United
States, Europe, and in other geographies ourselves or through
our partners. In addition, 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 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 submission of a supplemental new drug application to
the FDA for AYVAKIT for the treatment of indolent SM; plans and
timing for presenting detailed data from the PIONEER trial of
AYVAKIT in patients with non-advanced SM, and, expectations
regarding the potential benefits of AYVAKIT in treating patients
with non-advanced SM; statements regarding plans, strategies,
timelines and expectations for interactions with the FDA and other
regulatory authorities; statements regarding plans and expectations
for Blueprint Medicines' current or future approved drugs and drug
candidates; the potential benefits of any of Blueprint Medicines'
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; Blueprint Medicines' ability to realize the anticipated
benefits of its executive leadership transition plan; 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