-- For the first time, patients with indolent
SM have a medicine that treats the primary disease driver and is
proven to provide broad and durable symptom relief --
-- Approval based on the positive PIONEER
trial, in which once-daily AYVAKIT achieved significant
improvements in disease symptoms and measures of mast cell burden,
with a safety profile that compared favorably to placebo --
-- Blueprint Medicines to host investor
conference call and webcast today at 4:30
p.m. ET --
CAMBRIDGE, Mass., May 22, 2023
/PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC)
today announced that the U.S. Food and Drug
Administration (FDA) has approved AYVAKIT® (avapritinib)
for the treatment of adults with indolent systemic
mastocytosis (ISM). All U.S. patients with ISM now have access to
the first and only approved medicine designed to treat their
disease.
Systemic mastocytosis (SM) is a rare hematologic disorder that
can lead to a range of debilitating symptoms across multiple organ
systems and a significant impact on patients' quality of life. ISM
represents the vast majority of SM cases, and AYVAKIT is now
available for adults with ISM at the recommended dose of 25 mg once
daily. AYVAKIT was designed to potently and selectively inhibit KIT
D816V, the primary underlying driver of the disease. AYVAKIT has
been FDA approved for the treatment of advanced SM since
June 2021.
"Today's approval of AYVAKIT reflects more than a decade of
collaboration and leadership in the field of systemic mastocytosis,
during which our team at Blueprint Medicines has worked closely
with clinicians, patients and their families, and patient advocacy
groups to advance innovative science and develop a transformational
treatment for people living with the disease," said Becker
Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "AYVAKIT
is the first and only medicine approved by the FDA to treat
indolent systemic mastocytosis, marking a shift in the treatment
paradigm from supportive care to disease modifying therapy. In
addition, AYVAKIT is the only treatment approved across the
spectrum of indolent and advanced systemic mastocytosis. With a
broad indication for indolent systemic mastocytosis and a strong
label, we are now engaging healthcare providers to redefine what it
means for their patients to be well-controlled as well as
activating the patient community to seek out optimal care and
treatment."
"After decades of caring for people with indolent systemic
mastocytosis, I have seen firsthand its profound impact on
patients' underlying mast cell burden, symptoms, physical and
mental health, and ability to work and participate in daily
activities," said Cem Akin, M.D.,
Ph.D., Professor of Medicine at the University
of Michigan,1 and an investigator on the PIONEER
trial.1 "Despite the use of multiple supportive care
treatments, a considerable number of patients with indolent
systemic mastocytosis continue to experience a substantial disease
burden. AYVAKIT advances the treatment of indolent systemic
mastocytosis by targeting KIT D816V, the primary underlying cause
of the disease, and establishes a new standard of care for a broad
population of patients with this disorder. AYVAKIT delivered
statistically significant and consistent clinical improvements in
the PIONEER trial, and based on these practice-changing data, I
feel a tremendous sense of hope for the future for all those
affected by the disease."
The approval of AYVAKIT in ISM is based on data from the
double-blind, placebo-controlled PIONEER trial – the largest study
ever conducted for this disease – in which patients received
AYVAKIT 25 mg once daily plus best supportive care (AYVAKIT) or
placebo plus best supportive care (placebo). AYVAKIT demonstrated
significant improvements versus placebo in the primary and all key
secondary endpoints, including overall symptoms and measures of
mast cell burden.
AYVAKIT was well-tolerated with a favorable safety profile
compared to placebo, and most adverse reactions were mild to
moderate in severity. The most common adverse reactions for AYVAKIT
(≥10 percent) were eye edema, dizziness, peripheral edema and
flushing. Serious adverse reactions and discontinuations due to
adverse reactions occurred in less than 1 percent of patients.
Detailed results from the PIONEER trial, including open-label
extension study data showing the clinical benefits of AYVAKIT
through 48 weeks of treatment, were presented in February 2023 at the American Academy of Allergy,
Asthma & Immunology (AAAAI) Annual Meeting.
"People with indolent systemic mastocytosis are significantly
impacted by their disease symptoms, and many individuals
self-isolate at home to protect against unpredictable external
triggers. Ultimately, the disease can disrupt an individual's
quality of life, career and ability to interact with family and
friends, and those affected often face frequent healthcare visits
and significant polypharmacy burden," said Judith Kain Emmel, Board Chair of The Mast Cell
Disease Society. "Since we began nearly 30 years ago, our goals as
an organization have been to educate, advocate and collaborate for
the advancement of knowledge and research in mast cell disorders
like systemic mastocytosis. Today's approval is a historic moment
for the SM community and offers new hope for patients and their
families. We are honored to have contributed to this milestone by
facilitating SM community insights on the design of the PIONEER
trial and supporting the development of the validated symptom
assessment tool used in the study."
Blueprint Medicines is committed to patient access and
affordability for its approved medicines through YourBlueprint®,
which is designed to provide dedicated, personalized assistance for
eligible patients. Commercially insured patients who qualify for
the company's co-pay assistance program may pay as little as
$0 in out-of-pocket costs for
AYVAKIT. YourBlueprint offers additional programs intended to
facilitate timely access to therapy, including a patient assistance
program for eligible patients who have inadequate insurance
coverage. For more information,
visit YourBlueprint.com or call 1-888-BLUPRNT
(1-888-258-7768), Monday to Friday, 8:00 a.m. to 8:00 p.m. ET. Healthcare providers who
prescribe AYVAKIT can fill out an enrollment form at
YourBlueprint.com/HCP to help patients access Blueprint
Medicines' support programs.
Investor Conference Call Information
Blueprint Medicines will host a live conference call and
webcast today at 4:30 p.m. ET to discuss the FDA approval
of AYVAKIT in ISM. The conference call may be accessed by dialing
833-470-1428 (domestic) or 404-975-4839 (international), and
referring to conference ID 832860. A webcast of the call will also
be available under "Events and Presentations" in the Investors
& Media section of the Blueprint Medicines website at
https://ir.blueprintmedicines.com/. The archived webcast will be
available on the Blueprint Medicines website
approximately two hours after the conference call and will be
available for 30 days following the call.
About AYVAKIT (avapritinib)
AYVAKIT (avapritinib) is a precision therapy approved by the FDA
for the treatment of three indications: adults with ISM, 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. The vast majority of those affected have indolent systemic
mastocytosis (ISM). 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.
Historically, there had been no approved therapies for the
treatment of ISM.
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.
Important Safety Information
Intracranial Hemorrhage—Serious intracranial hemorrhage (ICH)
may occur with AYVAKIT treatment; fatal events occurred in <1%
of patients. Overall, ICH (eg, subdural hematoma, ICH, and cerebral
hemorrhage) occurred in 2.9% of 749 patients who received AYVAKIT
in clinical trials. In Advanced SM patients who received AYVAKIT at
200 mg daily, ICH occurred in 2 of 75 patients
(2.7%) who had platelet counts ≥50 x 109/L
prior to initiation of therapy and in
3 of 80 patients (3.8%) regardless of platelet
counts. In ISM patients, no events of ICH occurred in the
246 patients who received any dose of AYVAKIT in the PIONEER
study.
Monitor patients closely for risk factors of ICH which may
include history of vascular aneurysm, ICH or cerebrovascular
accident within the prior year, concomitant use of anticoagulant
drugs, or thrombocytopenia.
Symptoms of ICH may include headache, nausea, vomiting, vision
changes, or altered mental status. Advise patients to seek
immediate medical attention for signs or symptoms of ICH.
Permanently discontinue AYVAKIT if ICH of any grade occurs. In
Advanced SM patients, a platelet count must be performed prior to
initiating therapy. AYVAKIT is not recommended in Advanced SM
patients with platelet counts <50 x 109/L.
Following treatment initiation, platelet counts must be performed
every 2 weeks for the first 8 weeks. After 8 weeks of treatment,
monitor platelet counts every 2 weeks or as clinically indicated
based on platelet counts. Manage platelet counts of <50 x
109/L by treatment interruption or dose reduction.
Cognitive Effects—Cognitive adverse reactions can occur in
patients receiving AYVAKIT and occurred in 33% of 995 patients
overall in patients who received AYVAKIT in clinical trials
including: 28% of 148 Advanced SM patients (3% were
Grade ≥3), and 7.8% of patients with ISM who received
AYVAKIT + best supportive care (BSC) versus 7.0% of
patients who received placebo + BSC (<1% were
Grade 3). Depending on the severity and indication, withhold
AYVAKIT and then resume at same dose or at a reduced dose upon
improvement, or permanently discontinue.
Photosensitivity—AYVAKIT may cause photosensitivity
reactions. In all patients treated with AYVAKIT in clinical trials
(n=1049), photosensitivity reactions occurred in 2.5% of
patients. Advise patients to limit direct ultraviolet exposure
during treatment with AYVAKIT and for one week after
discontinuation of treatment.
Embryo-Fetal Toxicity—AYVAKIT can cause fetal harm when
administered to a pregnant woman. Advise pregnant women of the
potential risk to a fetus. Advise females and males of reproductive
potential to use an effective method of contraception during
treatment with AYVAKIT and for 6 weeks after the final dose of
AYVAKIT. Advise women not to breastfeed during treatment with
AYVAKIT and for 2 weeks after the final dose.
Adverse Reactions—The most common adverse reactions (≥20%)
in patients with Advanced SM were edema, diarrhea, nausea, and
fatigue/asthenia.
The most common adverse reactions (≥10%) in patients with
ISM were eye edema, dizziness, peripheral edema, and flushing.
Drug Interactions—Avoid coadministration of AYVAKIT with strong
or moderate CYP3A inhibitors. If coadministration with a moderate
CYP3A inhibitor cannot be avoided in patients with Advanced SM,
reduce dose of AYVAKIT. Avoid coadministration of AYVAKIT with
strong or moderate CYP3A inducers.
To report suspected adverse reactions, contact Blueprint
Medicines Corporation at 1-888-258-7768 or FDA at
1-800-FDA-1088 or http://www.fda.gov/medwatch.
Please click here to see the full Prescribing
Information for AYVAKIT.
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 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 FDA
approval of AYVAKIT/AYVAKYT and the implications of such approval
for patients, caregivers and healthcare professionals; expectations
regarding patients' ability to rapidly access treatment with
AYVAKIT/AYVAKYT; Blueprint Medicines' plans and ability to provide
robust support services for patients prescribed AYVAKIT/AYVAKYT
through YourBlueprint; the potential benefits of Blueprint
Medicines' current and future approved drugs or drug candidates in
treating patients, including expectations regarding the potential
of AYVAKIT/AYVAKYT to address additional tumor types and treatment
settings; 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 our ability and plans in continuing to
expand Blueprint Medicines' 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 our 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; 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 its products or any drug candidates it is
developing; Blueprint Medicines' ability to develop and
commercialize companion diagnostic tests for its products or any of
its current and future drug candidates; Blueprint Medicines'
ability to successfully expand its research platform and the costs
thereof; 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 its
Annual Report on Form 10-K for the year ended December 31, 2022, as filed with the SEC on
February 16, 2023, 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 assumes no obligation to update or revise these
forward-looking statements for any reason, even if new information
becomes available in the future.
Reference
1 Title provided for identification purposes
only. The views and opinions expressed are those of the individual
only and do not necessarily reflect the positions of the
University of Michigan. Dr. Akin has a
paid consulting/advisory relationship with Blueprint Medicines
Corporation.
Trademarks
Blueprint Medicines, AYVAKIT, AYVAKYT, YourBlueprint and
associated logos are trademarks of Blueprint Medicines
Corporation.
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SOURCE Blueprint Medicines Corporation