CAMBRIDGE, Mass., Jan. 28, 2022 /PRNewswire/ -- Blueprint
Medicines Corporation (NASDAQ: BPMC) today announced that the
Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency has adopted a positive opinion,
recommending to expand the current indication for AYVAKYT®
(avapritinib) to include monotherapy for the treatment of adult
patients 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.
"The positive opinion from the CHMP is a result of nearly a
decade of collaboration with the systemic mastocytosis community
and a dedication to bring a transformative therapy to treat and
manage this rare, life-threatening disease," said Becker Hewes,
M.D., Chief Medical Officer at Blueprint Medicines. "Today's
announcement brings patients with advanced forms of SM in
Europe one step closer to
accessing AYVAKYT, the first specifically designed precision
therapy to selectively target the primary driver of their disease.
Blueprint Medicines remains committed to improving outcomes for
patients living with systemic mastocytosis around the world."
"Patients across Europe are
waiting for innovative treatment options for advanced systemic
mastocytosis, which is associated with organ damage due to mast
cell proliferation and poor survival outcomes," said Prof. Dr.
Andreas Reiter, M.D., Center of
Excellence for Myeloproliferative Neoplasms, University Medicine
Mannheim. "A precision therapy associated with potent and selective
targeting of the KIT D816V mutation brings the prospect of changing
the course of disease and transforming treatment for patients to
set a new standard of care in Europe."
The CHMP opinion will now be reviewed by the European
Commission, which has the authority to grant and expand marketing
authorizations for medicinal products in the European Union. A
final decision on the application to expand the indication for
AYVAKYT is anticipated by early April
2022.
The CHMP based its opinion on results from the Phase 1 EXPLORER
trial and Phase 2 PATHFINDER trial, in which AYVAKYT showed durable
clinical efficacy in advanced SM patients across disease subtypes
and a generally well-tolerated safety profile. These two studies,
which enrolled approximately 150 patients with up to four years of
follow-up and comprise the largest ever reported dataset in
advanced systemic mastocytosis, were recently published in
Nature Medicine.
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. Under the brand name AYVAKIT, the medicine is approved in
the U.S. for the treatment of adults with unresectable or
metastatic GIST harboring a PDGFRA exon 18 mutation, including
PDGFRA D842V mutations, and for the treatment of adults with
Advanced SM, including aggressive SM (ASM), SM-AHN and mast cell
leukemia (MCL).1 It is also approved under the
brand name AYVAKIT in Mainland China for the treatment of adults
with unresectable or metastatic GIST harboring a PDGFRA exon 18
mutation, including PDGFRA D842V mutations, and in Hong Kong and Taiwan for the treatment of adults with
unresectable or metastatic GIST harboring a PDGFRA D842V
mutation.2-4
AYVAKYT/AYVAKIT is not approved for the treatment of any other
indication in the Europe, U.S., or Greater China, or
for any indication in any other jurisdiction by any other health
authority.
Blueprint Medicines is developing AYVAKYT/AYVAKIT globally
for the treatment of advanced and non-advanced SM. The European
Commission granted orphan medicinal product designation for AYVAKYT
for the treatment of GIST and mastocytosis. The U.S. Food and Drug
Administration (FDA) granted breakthrough therapy designation to
AYVAKIT for the treatment of moderate to severe indolent
SM.
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. 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 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. In Europe, there are about 40,000 patients with
SM, and advanced SM represents about 5 to 10 percent of this
patient population.5
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. Historically, there had been no approved therapies for
the treatment of SM that selectively inhibit D816V mutant
KIT.6,7
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 approved medicines
directly to patients in the United
States and Europe, and we
are globally advancing multiple programs for systemic mastocytosis,
lung 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 Blueprint Medicines' views with respect to the approval
of AYVAKYT and the implications of such approval for patients,
caregivers and healthcare professionals; expectations regarding the
potential benefits of AYVAKYT in treating patients with advanced
SM; expectations concerning when AYVAKYT will be commercially
available in Europe; 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 establishing a commercial infrastructure, and
successfully launching, marketing and selling current or future
approved products, including AYVAKIT and GAVRETO® (pralsetinib);
Blueprint Medicines' ability to successfully expand the approved
indications for AYVAKIT and GAVRETO or obtain marketing approval
for AYVAKIT and GAVRETO 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 develop and commercialize companion
diagnostic tests for its current and future drug candidates; and
the success of Blueprint Medicines' current and future
acquisitions, 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.
References
1 Blueprint Medicines.
AYVAKIT (avapritinib) Prescribing Information. Accessed at
https://www.blueprintmedicines.com/wp-content/uploads/uspi/AYVAKIT.pdf.
January 18, 2022.
2 CStone Announces China NMPA New Drug Approval of
Precision Therapy AYVAKIT® (avapritinib) for the Treatment of
Adults with Unresectable or Metastatic PDGFRA Exon 18 Mutant
Gastrointestinal Stromal Tumor. CStone Pharmaceuticals. Accessed at
https://www.cstonepharma.com/en/html/news/2573.html. January 26, 2022.
3 "CStone announced new drug approval of precision
therapy AYVAKIT® (avapritinib) in Hong
Kong, China for the treatment of PDGFRA D842V mutant
gastrointestinal stromal tumors (GIST)." CStone Pharmaceuticals.
Accessed at https://www.cstonepharma.com/en/html/news/2685.html.
January 26, 2022.
4 " CStone Announces Acceptance of New Drug
Application in Hong Kong for
Avapritinib for the Treatment of Adults with Unresectable or
Metastatic PDGFRA D842V Mutant Gastrointestinal Stromal Tumor."
Accessed at: https://www.cstonepharma.com/en/html/news/2586.html.
January 26, 2022.
5 Estimated SM prevalence and patient subtypes based on
internal claims analysis and epidemiology reported in Orphanet
(orpha.net) and Cohen SS, Skovbo S, Vestergaard H, et al.
Epidemiology of systemic mastocytosis in Denmark. Br J Haemotol.
2014;166(4):521-528.
6 Jennings SV, Slee VM, Zack RM, et al. Patient
perceptions in mast cell disorders. Immunol Allergy Clin North Am.
2018;38(3):505-525.
7 Mesa, RA, Sullivan, EM, Dubinski, D, et al. Patient
reported outcomes among systemic mastocytosis (SM) patients in
routine clinical practice: results from the TouchStone Survey.
Blood. 2020;136(1):37.
Trademarks
Blueprint Medicines AYVAKYT, AYVAKIT,
GAVRETO and associated logos are trademarks of Blueprint Medicines
Corporation.
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SOURCE Blueprint Medicines Corporation