-- In the European Union,
patients with indolent systemic mastocytosis now have an approved
medicine that treats the primary driver of
disease --
-- Approval based on data from
PIONEER trial, in which AYVAKYT achieved significant improvements
across a broad range of symptoms with a safety profile comparable
to placebo1 --
CAMBRIDGE, Mass., Dec. 12,
2023 /PRNewswire/ -- Blueprint Medicines
Corporation (Nasdaq: BPMC) today announced the European Commission
has approved AYVAKYT® (avapritinib) for the treatment of
adult patients with indolent systemic mastocytosis (ISM) with
moderate to severe symptoms inadequately controlled on symptomatic
treatment. AYVAKYT is the first and only approved therapy for
people living with ISM in Europe.
Systemic mastocytosis (SM) is a rare hematologic disorder that
can lead to a range of debilitating symptoms with a significant
impact on patients' quality of life. The majority of patients
living with SM have ISM, and there are approximately 40,000 people
living with ISM in the European Union.2,3* AYVAKYT
was designed to potently and selectively target KIT D816V, the
primary underlying driver of the disease.
"Today's approval represents an important step toward
delivering a new global standard of care for patients with ISM and
builds on years of collaboration with the SM community," said
Georg Pirmin Meyer, M.D., Senior
Vice President, International at Blueprint Medicines. "For the
first time in Europe ISM patients have an approved therapy, marking
a new era in the treatment of this disease. AYVAKYT is the first
approved medicine for both ISM and advanced SM, and our team is
committed to bringing this transformative therapy to patients
across the spectrum of disease."
"Indolent systemic mastocytosis can be characterized by
significant symptom burden across multiple organ systems, which can
profoundly impact patients' ability to perform activities of daily
living in a relevant proportion of patients," said Jens Panse, M.D., Deputy Director of the
Department of Hematology/Oncology of the University Hospital RWTH
Aachen. "AYVAKYT represents an important treatment
breakthrough as the first medicine approved for patients living
with ISM, and the only therapy designed to selectively target the
primary genetic driver of the disease. In the PIONEER trial,
AYVAKYT showed statistically significant and durable clinical
benefits across all measured ISM symptoms with a well-tolerated
safety profile. Based on these practice-changing data, AYVAKYT has
the potential to advance treatment for a broad range of patients
living with ISM."
The approval follows the positive opinion by the Committee for
Medicinal Products for Human Use (CHMP), and this EC decision is
based on data from the double-blind, placebo-controlled PIONEER
trial – the largest study ever conducted in ISM. AYVAKYT
showed clinically meaningful improvements versus placebo in the
primary and all key secondary endpoints, including overall symptoms
and measures of mast cell burden. AYVAKYT was well-tolerated with a
favorable safety profile, and most adverse events (AEs) were
reported as mild (Grade 1). The most common AEs were flushing,
edema, increased blood alkaline phosphate and
insomnia.1
"Many people living with indolent systemic mastocytosis face
unpredictable and severe symptoms, which significantly impair their
ability to work or spend quality time with their family, friends
and communities," said Patrizia
Marcis, President of the Associazione Italiana Mastocitosi
(ASIMAS) ODV. "Today's approval offers a new sense of hope to the
ISM community, and we are proud to collaborate with clinical
researchers, patients and companies like Blueprint Medicines to
advance care for all those living with the disease."
In Europe, Blueprint Medicines
plans to initiate its first commercial launch in Germany, followed by additional markets based
on local healthcare technology assessment and reimbursement process
timelines.
About AYVAKYT®
(avapritinib)
AYVAKYT® (avapritinib) is a kinase inhibitor approved by the
European Commission for the treatment of three indications: adults
with indolent systemic mastocytosis (ISM), adults with aggressive
systemic mastocytosis (ASM), systemic mastocytosis with an
associated hematological neoplasm (SM-AHN) or mast cell leukemia
(MCL), after at least one systemic therapy, and adults with
unresectable or metastatic gastrointestinal stromal tumors (GIST)
harboring the PDGFRA D842V mutation.4 Under the
brand name AYVAKIT®, the medicine is approved in the
U.S. for the treatment of adults with ISM, adults with advanced SM,
including ASM, SM-AHN and MCL, and adults with unresectable or
metastatic GIST harboring a PDGFRA exon 18 mutation, including
PDGFRA D842V mutations.5
To learn about ongoing or planned clinical trials,
contact Blueprint Medicines at
medinfoeurope@blueprintmedicines.com and +31 85 064 4001.
Additional information is available at blueprintclinicaltrials.com
and clinicaltrials.gov.
Please click here to see the Summary of Product Characteristics
for AYVAKYT.
About Systemic Mastocytosis
Systemic mastocytosis (SM) is a rare disease driven by the KIT
D816V mutation in about 95 percent of cases.6
Uncontrolled proliferation and activation of mast cells result in
chronic, severe and often unpredictable symptoms for patients
across the spectrum of SM. In the European Union, approximately
40,000 people live with indolent systemic
mastocytosis.2,3* A broad range of symptoms, including
anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog,
fatigue and bone pain, frequently persist in patients with ISM
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.
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 have brought our approved medicines
to patients in the United States
and Europe, and we are globally
advancing multiple programs for mast cell disorders, including
systemic mastocytosis and chronic urticaria, breast cancer and
other cancers vulnerable to CDK2 inhibition, as well as EGFR-mutant
lung cancer. 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 Blueprint Medicines' views with respect to the
implications of the approval of AYVAKYT for people living with ISM
in Europe; plans for Blueprint
Medicines' first commercial launch in Germany, followed by additional markets;
plans, strategies, timelines 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: preliminary activity and safety data may not be
representative of more mature data; the risk of delay of any
current or planned clinical trials or the development of Blueprint
Medicines' current or future drug candidates; risks related to
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; preclinical and clinical
results for Blueprint Medicines' drug candidates 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 may be delayed or slower than
anticipated; actions of regulatory agencies may affect the
initiation, timing and progress of clinical trials; the success of
Blueprint Medicines' current and future collaborations, financing
arrangements, partnerships or licensing arrangements may impact
Blueprint Medicines' ability to capitalize on the market potential
of its approved drugs and drug candidates; and risks related to
Blueprint Medicines' ability to obtain, maintain and enforce patent
and other intellectual property protection for its products and
current or future drug candidates it is developing. 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.
References
*Based on Cohen 2014 study of 548 adults with SM diagnosed from
1997 to 2010 in linked Danish national health registries, with a
14-year limited-duration prevalence estimated at 9.59 per 100,000
as of 1 January 2011
- Gotlib J, et al. Avapritinib versus Placebo in Indolent
Systemic Mastocytosis. NEJM Evid 2023;2(6) May 23, 2023.
- Cohen SS et al. Epidemiology of systemic mastocytosis in
Denmark. Br J Haematol.
2014;166(4):521-8.
- OrphaNet. Indolent systemic mastocytosis. Available
at: https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=en&Expert=98848.
Accessed December 2023.
- AYVAKYT SMPC. Available at:
https://www.ema.europa.eu/en/documents/
product-information/ayvakyt-epar-product-information_en.pdf.
Accessed December 2023.
- Blueprint Medicines. AYVAKIT (avapritinib) Prescribing
Information. Available at:
https://www.blueprintmedicines.com/wp-content/uploads/uspi/AYVAKIT.pdf.
Accessed December 2023
- Mesa R.A et al. Perceptions of patient disease burden and
management approaches in systemic mastocytosis: Results of the
TouchStone Healthcare Provider Survey. Cancer.
2022;128(20):3700-3708
Trademarks
Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are
trademarks of Blueprint Medicines Corporation.
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