CAMBRIDGE, Mass., March 3, 2021 /PRNewswire/ -- Blueprint Medicines
Corporation (NASDAQ: BPMC) today announced that the European
Medicines Agency (EMA) has validated the company's Type II
variation marketing authorization application for AYVAKYT®
(avapritinib) for the treatment of advanced systemic mastocytosis
(SM). Validation of the application confirms that the submission is
sufficiently complete to begin the formal review process. The
European Commission has granted orphan medicinal product
designation to AYVAKYT for the treatment of mastocytosis.
"AYVAKYT represents a promising approach to address the needs of
patients with advanced systemic mastocytosis, a rare,
life-threatening disease characterized by severe organ damage,"
said Paul Beresford, Senior Vice
President and General Manager, International, at Blueprint
Medicines. "In robust datasets across our registrational trials,
AYVAKYT has shown complete and durable remissions not typically
observed in this patient population, and a generally well-tolerated
safety profile. We look forward to working closely with the EMA
during the review process, and aim to bring the first precision
therapy to patients in Europe that
targets the primary driver of the disease."
AYVAKYT, a potent and selective inhibitor of D816V mutant KIT,
is being developed to treat advanced and non-advanced forms of SM.
Last month, Blueprint Medicines announced that the U.S. Food and
Drug Administration (FDA) accepted the company's supplemental new
drug application and granted priority review for AYVAKIT™
(avapritinib) for the treatment of advanced SM.
About SM
SM is a rare disease driven by the KIT D816V mutation.
Uncontrolled proliferation and activation of mast cells result in
chronic, severe and often unpredictable symptoms for patients
across the spectrum of SM. The vast majority of those affected have
non-advanced (indolent or smoldering) SM, with debilitating
symptoms that lead to a profound, negative impact on quality of
life. A minority of patients have advanced SM, which encompasses a
group of high-risk SM subtypes including aggressive SM, SM
with an associated hematological neoplasm and mast cell leukemia.
In addition to mast cell activation symptoms, advanced SM is
associated with organ damage due to mast cell infiltration and poor
survival.
Debilitating symptoms, including anaphylaxis, maculopapular
rash, pruritis, diarrhea, brain fog, fatigue and bone pain, often
persist across all forms of SM despite treatment with a number of
symptomatic therapies. 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 SM that selectively inhibit D816V mutant KIT.
About AYVAKYT (avapritinib)
AYVAKYT (avapritinib) is a kinase inhibitor approved by the
European Commission for the treatment of adults with unresectable
or metastatic gastrointestinal stromal tumors (GIST) harboring the
PDGFRA D842V mutation. This medicine was approved by the FDA for
the treatment of adults with unresectable or metastatic GIST
harboring a PDGFRA exon 18 mutation, including PDGFRA D842V
mutations, under the brand name AYVAKIT.
AYVAKIT/AYVAKYT is not approved for the treatment of any other
indication, including SM, in the U.S. by the FDA or in Europe by the European Commission, or for any
indication in any other jurisdiction by any other health
authority.
Blueprint Medicines is developing AYVAKIT globally for the
treatment of advanced and indolent SM. The FDA granted breakthrough
therapy designation to AYVAKIT for the treatment of advanced SM,
including the subtypes of aggressive SM, SM with an associated
hematological neoplasm and mast cell leukemia, and for the
treatment of moderate to severe indolent SM.
Blueprint Medicines has an exclusive collaboration and license
agreement with CStone Pharmaceuticals for the development and
commercialization of AYVAKIT in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains
development and commercial rights for AYVAKIT in the rest of the
world.
About Blueprint Medicines
Blueprint Medicines is a global precision therapy company
that invents life-changing medicines for people with cancer and
hematologic disorders. Applying an approach that is both precise
and agile, we create therapies 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 genomically defined cancers, systemic mastocytosis,
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 potential benefits of AYVAKIT/AYVAKYT in treating
patients with SM; plans, timelines and expectations for
interactions with the EMA; plans, timelines and expectations for
the commercialization of AYVAKYT for the treatment of advanced SM,
if approved by the European Commission; 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 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 and GAVRETO™ (pralsetinib) 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; 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, GAVRETO or
any drug candidates it is developing; Blueprint Medicines' ability
to develop and commercialize companion diagnostic tests for
AYVAKIT/AYVAKYT, GAVRETO or any of its current and future drug
candidates; and the success of Blueprint Medicines' current and
future collaborations, 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, GAVRETO and associated
logos are trademarks of Blueprint Medicines Corporation.
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SOURCE Blueprint Medicines Corporation