BRIDGEWATER, N.J., March 23, 2021 /PRNewswire/ -- Insmed
Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a
mission to transform the lives of patients with serious and rare
diseases, today announced that Japan's Ministry of Health, Labour and Welfare
(MHLW) has approved ARIKAYCE® (amikacin liposome
inhalation suspension) for the treatment of patients with
nontuberculous mycobacterial (NTM) lung disease caused by
Mycobacterium avium complex (MAC) who did not sufficiently
respond to prior treatment with a multidrug regimen (MDR). The
launch of ARIKAYCE in Japan is
anticipated in mid-2021.
"Today's approval of ARIKAYCE is a significant milestone for
patients with refractory MAC lung disease in Japan, who currently have no approved
treatments available specifically for this serious and chronic
pulmonary disease. It is also the realization of Insmed's long-term
commitment to building our business in Japan, and a meaningful step in our efforts to
bring ARIKAYCE to patients with refractory MAC lung disease around
the world," said Will Lewis, Chair
and Chief Executive Officer of Insmed. "We are excited by the
opportunity to serve patients in Japan, where the burden of this disease is
particularly high."
The approval by the MHLW is based on results from the
randomized, open-label, global Phase 3 CONVERT study, which
demonstrated that once-daily ARIKAYCE, when combined with MDR,
improved sputum culture conversion rates in patients with
refractory NTM lung disease caused by MAC compared to MDR therapy
alone. The most common side effects with ARIKAYCE affecting the
respiratory system are dysphonia, cough, dyspnea, and
hemoptysis.
"For many patients living with MAC lung disease, standard
treatment is inadequate, leaving symptoms to potentially worsen
over time," said Yuji Orihara,
General Manager, Japan, Insmed.
"The need for new and effective approaches is clear and we look
forward to making ARIKAYCE available for appropriate patients in
Japan in the coming months."
ARIKAYCE is administered using the Lamira® Nebulizer
System, which was authorized for use in Japan by the MHLW in June of 2020. As part of
Insmed's comprehensive approach to patient care, a support program
will be available at the time of launch to provide support and
information to patients in Japan
who are prescribed ARIKAYCE.
ARIKAYCE is approved in the United
States for the treatment of MAC lung disease as part of a
combination antibacterial drug regimen for adult patients with
limited or no alternative treatment options in a refractory
setting, and in Europe (under the
name ARIKAYCE® Liposomal 590 mg Nebuliser Dispersion)
for the treatment of NTM lung infections caused by MAC in adults
with limited treatment options who do not have cystic fibrosis.
About MAC Lung Disease
Mycobacterium avium complex (MAC) lung disease is a
chronic and serious disorder that can significantly increase
morbidity and mortality. Patients with MAC lung disease can
experience a range of symptoms that often worsen over time,
including chronic cough, dyspnea, fatigue, fever, weight loss, and
chest pain. In some cases, MAC lung disease can cause severe, even
permanent damage to the lungs, and can be fatal. MAC lung disease
is an emerging public health concern worldwide with significant
unmet need.
About ARIKAYCE
ARIKAYCE is approved in the United States as
ARIKAYCE® (amikacin liposome inhalation
suspension), in Europe as
ARIKAYCE® Liposomal 590 mg Nebuliser Dispersion,
and in Japan as
ARIKAYCE® inhalation 590 mg (amikacin sulfate inhalation
drug product). Current international treatment guidelines recommend
the use of ARIKAYCE for appropriate patients. ARIKAYCE is a novel,
inhaled, once-daily formulation of amikacin, an established
antibiotic that was historically administered intravenously and
associated with severe toxicity to hearing, balance, and kidney
function. Insmed's proprietary PULMOVANCE™ liposomal technology
enables the delivery of amikacin directly to the lungs, where
liposomal amikacin is taken up by lung macrophages where the
infection resides, while limiting systemic exposure. ARIKAYCE is
administered once daily using the Lamira® Nebulizer
System manufactured by PARI Pharma GmbH (PARI).
About PARI Pharma and the Lamira® Nebulizer
System
ARIKAYCE is delivered by a novel inhalation device, the
Lamira® Nebulizer System, developed by PARI.
Lamira® is a quiet, portable nebulizer that enables
efficient aerosolization of ARIKAYCE via a vibrating, perforated
membrane. Based on PARI's 100-year history working with aerosols,
PARI is dedicated to advancing inhalation therapies by developing
innovative delivery platforms to improve patient care.
IMPORTANT SAFETY INFORMATION FOR ARIKAYCE IN THE U.S.
WARNING: RISK OF
INCREASED RESPIRATORY ADVERSE REACTIONS
ARIKAYCE has been
associated with an increased risk of respiratory adverse
reactions,
including hypersensitivity pneumonitis, hemoptysis, bronchospasm,
and exacerbation of
underlying pulmonary disease that have led to hospitalizations in
some cases.
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Hypersensitivity Pneumonitis has been reported with the
use of ARIKAYCE in the clinical trials. Hypersensitivity
pneumonitis (reported as allergic alveolitis, pneumonitis,
interstitial lung disease, allergic reaction to ARIKAYCE) was
reported at a higher frequency in patients treated with ARIKAYCE
plus background regimen (3.1%) compared to patients treated with a
background regimen alone (0%). Most patients with hypersensitivity
pneumonitis discontinued treatment with ARIKAYCE and received
treatment with corticosteroids. If hypersensitivity pneumonitis
occurs, discontinue ARIKAYCE and manage patients as medically
appropriate.
Hemoptysis has been reported with the use of ARIKAYCE in
the clinical trials. Hemoptysis was reported at a higher frequency
in patients treated with ARIKAYCE plus background regimen (17.9%)
compared to patients treated with a background regimen alone
(12.5%). If hemoptysis occurs, manage patients as medically
appropriate.
Bronchospasm has been reported with the use of
ARIKAYCE in the clinical trials. Bronchospasm (reported as asthma,
bronchial hyperreactivity, bronchospasm, dyspnea, dyspnea
exertional, prolonged expiration, throat tightness, wheezing) was
reported at a higher frequency in patients treated with ARIKAYCE
plus background regimen (28.7%) compared to patients treated
with a background regimen alone (10.7%). If bronchospasm occurs
during the use of ARIKAYCE, treat patients as medically
appropriate.
Exacerbations of underlying pulmonary disease has
been reported with the use of ARIKAYCE in the clinical trials.
Exacerbations of underlying pulmonary disease (reported as chronic
obstructive pulmonary disease (COPD), infective exacerbation of
COPD, infective exacerbation of bronchiectasis) have been reported
at a higher frequency in patients treated with ARIKAYCE plus
background regimen (14.8%) compared to patients treated with
background regimen alone (9.8%). If exacerbations of
underlying pulmonary disease occur during the use of ARIKAYCE,
treat patients as medically appropriate.
Anaphylaxis and Hypersensitivity Reactions: Serious
and potentially life-threatening hypersensitivity reactions,
including anaphylaxis, have been reported in patients taking
ARIKAYCE. Signs and symptoms include acute onset of skin and
mucosal tissue hypersensitivity reactions (hives, itching,
flushing, swollen lips/tongue/uvula), respiratory difficulty
(shortness of breath, wheezing, stridor, cough), gastrointestinal
symptoms (nausea, vomiting, diarrhea, crampy abdominal pain), and
cardiovascular signs and symptoms of anaphylaxis (tachycardia, low
blood pressure, syncope, incontinence, dizziness). Before therapy
with ARIKAYCE is instituted, evaluate for previous hypersensitivity
reactions to aminoglycosides. If anaphylaxis or a hypersensitivity
reaction occurs, discontinue ARIKAYCE and institute appropriate
supportive measures.
Ototoxicity has been reported with the use of ARIKAYCE in
the clinical trials. Ototoxicity (including deafness, dizziness,
presyncope, tinnitus, and vertigo) were reported with a higher
frequency in patients treated with ARIKAYCE plus background regimen
(17%) compared to patients treated with background
regimen alone (9.8%). This was primarily driven by tinnitus
(7.6% in ARIKAYCE plus background regimen vs 0.9% in the background
regimen alone arm) and dizziness (6.3% in ARIKAYCE plus background
regimen vs 2.7% in the background regimen alone arm). Closely
monitor patients with known or suspected auditory or vestibular
dysfunction during treatment with ARIKAYCE. If ototoxicity occurs,
manage patients as medically appropriate, including potentially
discontinuing ARIKAYCE.
Nephrotoxicity was observed during the clinical
trials of ARIKAYCE in patients with MAC lung disease but not at a
higher frequency than background regimen alone. Nephrotoxicity has
been associated with the aminoglycosides. Close monitoring of
patients with known or suspected renal dysfunction may be needed
when prescribing ARIKAYCE.
Neuromuscular Blockade: Patients with neuromuscular
disorders were not enrolled in ARIKAYCE clinical trials. Patients
with known or suspected neuromuscular disorders, such as myasthenia
gravis, should be closely monitored since aminoglycosides may
aggravate muscle weakness by blocking the release of acetylcholine
at neuromuscular junctions.
Embryo-Fetal Toxicity: Aminoglycosides can cause
fetal harm when administered to a pregnant woman. Aminoglycosides,
including ARIKAYCE, may be associated with total, irreversible,
bilateral congenital deafness in pediatric patients exposed in
utero. Patients who use ARIKAYCE during pregnancy, or become
pregnant while taking ARIKAYCE should be apprised of the potential
hazard to the fetus.
Contraindications: ARIKAYCE is contraindicated in
patients with known hypersensitivity to any aminoglycoside.
Most Common Adverse Reactions: The most common adverse
reactions in Trial 1 at an incidence ≥5% for patients using
ARIKAYCE plus background regimen compared to patients treated with
background regimen alone were dysphonia (47% vs 1%), cough (39% vs
17%), bronchospasm (29% vs 11%), hemoptysis (18% vs 13%),
ototoxicity (17% vs 10%), upper airway irritation (17% vs 2%),
musculoskeletal pain (17% vs 8%), fatigue and asthenia (16% vs
10%), exacerbation of underlying pulmonary disease (15% vs 10%),
diarrhea (13% vs 5%), nausea (12% vs 4%), pneumonia (10% vs 8%),
headache (10% vs 5%), pyrexia (7% vs 5%), vomiting (7% vs 4%), rash
(6% vs 2%), decreased weight (6% vs 1%), change in sputum (5% vs
1%), and chest discomfort (5% vs 3%).
Drug Interactions: Avoid concomitant use of ARIKAYCE
with medications associated with neurotoxicity, nephrotoxicity, and
ototoxicity. Some diuretics can enhance aminoglycoside toxicity by
altering aminoglycoside concentrations in serum and tissue. Avoid
concomitant use of ARIKAYCE with ethacrynic acid, furosemide, urea,
or intravenous mannitol.
Overdosage: Adverse reactions specifically associated
with overdose of ARIKAYCE have not been identified. Acute
toxicity should be treated with immediate withdrawal of ARIKAYCE,
and baseline tests of renal function should be undertaken.
Hemodialysis may be helpful in removing amikacin from the body. In
all cases of suspected overdosage, physicians should contact the
Regional Poison Control Center for information about effective
treatment.
U.S. INDICATION
LIMITED POPULATION: ARIKAYCE is indicated in adults, who
have limited or no alternative treatment options, for the treatment
of Mycobacterium avium complex (MAC) lung disease as
part of a combination antibacterial drug regimen in patients who do
not achieve negative sputum cultures after a minimum of 6
consecutive months of a multidrug background regimen therapy. As
only limited clinical safety and effectiveness data for ARIKAYCE
are currently available, reserve ARIKAYCE for use in adults who
have limited or no alternative treatment options. This drug
is indicated for use in a limited and specific population of
patients.
This indication is approved under accelerated approval based
on achieving sputum culture conversion (defined as 3 consecutive
negative monthly sputum cultures) by Month 6. Clinical benefit has
not yet been established. Continued approval for this indication
may be contingent upon verification and description of clinical
benefit in confirmatory trials.
Limitation of Use: ARIKAYCE has only been studied
in patients with refractory MAC lung disease defined as patients
who did not achieve negative sputum cultures after a minimum of 6
consecutive months of a multidrug background regimen therapy. The
use of ARIKAYCE is not recommended for patients with non-refractory
MAC lung disease.
Patients are encouraged to report negative side effects of
prescription drugs to the FDA.
Visit www.fda.gov/medwatch, or call 1–800–FDA–1088. You
can also call the Company at 1-844-4-INSMED.
Please see Full Prescribing
Information.
About Insmed
Insmed Incorporated is a global biopharmaceutical company on a
mission to transform the lives of patients with serious and rare
diseases. Insmed's first commercial product is a first-in-disease
therapy approved in the United
States, Europe, and
Japan to treat a chronic,
debilitating lung disease. The Company is also progressing a robust
pipeline of investigational therapies targeting areas of serious
unmet need, including neutrophil-mediated inflammatory diseases and
rare pulmonary disorders. Insmed is headquartered in Bridgewater, New Jersey, with a growing
footprint across Europe and in
Japan. For more information, visit
www.insmed.com.
Forward-looking Statements
This press release contains forward-looking statements that
involve substantial risks and uncertainties. "Forward-looking
statements," as that term is defined in the Private Securities
Litigation Reform Act of 1995, are statements that are not
historical facts and involve a number of risks and uncertainties.
Words herein such as "may," "will," "should," "could," "would,"
"expects," "plans," "anticipates," "believes," "estimates,"
"projects," "predicts," "intends," "potential," "continues," and
similar expressions (as well as other words or expressions
referencing future events, conditions or circumstances) may
identify forward-looking statements.
The forward-looking statements in this press release are based
upon the Company's current expectations and beliefs, and involve
known and unknown risks, uncertainties and other factors, which may
cause the Company's actual results, performance and achievements
and the timing of certain events to differ materially from the
results, performance, achievements or timing discussed, projected,
anticipated or indicated in any forward-looking statements. Such
risks, uncertainties and other factors include, among others, the
following: failure to obtain, or delays in obtaining, regulatory
approvals for ARIKAYCE outside the U.S., Europe or Japan, or for the Company's product candidates
in the U.S., Europe, Japan or other markets; failure
to successfully commercialize ARIKAYCE, the Company's only approved
product, in the U.S., Europe or
Japan (amikacin liposome
inhalation suspension, Liposomal 590 mg Nebuliser Dispersion or
amikacin sulfate inhalation drug product, respectively), or to
maintain U.S., European or Japanese approval for ARIKAYCE; business
or economic disruptions due to catastrophes or other events,
including natural disasters or public health crises; impact of the
novel coronavirus (COVID-19) pandemic and efforts to reduce its
spread on the Company's business, employees, including key
personnel, patients, partners and suppliers; risk that brensocatib
does not prove effective or safe for patients in ongoing and future
clinical studies, including the ASPEN study; risk that TPIP does not prove to
be effective or safe for patients in ongoing and future clinical
studies; uncertainties in the degree of market acceptance of
ARIKAYCE by physicians, patients, third-party payors and others in
the healthcare community; the Company's inability to obtain full
approval of ARIKAYCE from the U.S. Food and Drug Administration,
including the risk that the Company will not timely and
successfully complete the study to validate a PRO tool and the
confirmatory post-marketing study required for full approval of
ARIKAYCE; inability of the Company, PARI Pharma GmbH (PARI) or the
Company's other third-party manufacturers to comply with regulatory
requirements related to ARIKAYCE or the
Lamira® Nebulizer System; the Company's inability
to obtain adequate reimbursement from government or third-party
payors for ARIKAYCE or acceptable prices for ARIKAYCE; development
of unexpected safety or efficacy concerns related to ARIKAYCE or
the Company's product candidates; inaccuracies in the Company's
estimates of the size of the potential markets for ARIKAYCE or its
product candidates or in data the Company has used to identify
physicians, expected rates of patient uptake, duration of expected
treatment, or expected patient adherence or discontinuation rates;
the Company's inability to create an effective direct sales and
marketing infrastructure or to partner with third parties that
offer such an infrastructure for distribution of ARIKAYCE or any of
the Company's product candidates that are approved in the future;
failure to obtain regulatory approval to expand ARIKAYCE's
indication to a broader patient population; failure to successfully
conduct future clinical trials for ARIKAYCE, brensocatib, TPIP and
the Company's other product candidates due to the Company's limited
experience in conducting preclinical development activities and
clinical trials necessary for regulatory approval and its potential
inability to enroll or retain sufficient patients to conduct and
complete the trials or generate data necessary for regulatory
approval, among other things; risks that the Company's clinical
studies will be delayed or that serious side effects will be
identified during drug development; failure of third parties on
which the Company is dependent to manufacture sufficient quantities
of ARIKAYCE or the Company's product candidates for commercial or
clinical needs, to conduct the Company's clinical trials, or to
comply with the Company's agreements or laws and regulations that
impact the Company's business or agreements with the Company; the
Company's inability to attract and retain key personnel or to
effectively manage the Company's growth; the Company's inability to
adapt to its highly competitive and changing environment; the
Company's inability to adequately protect its intellectual property
rights or prevent disclosure of its trade secrets and other
proprietary information and costs associated with litigation or
other proceedings related to such matters; restrictions or other
obligations imposed on the Company by its agreements related to
ARIKAYCE or the Company's product candidates, including its license
agreements with PARI and AstraZeneca AB, and failure of the Company
to comply with its obligations under such agreements; the cost and
potential reputational damage resulting from litigation to which
the Company is or may become a party, including product liability
claims; the Company's limited experience operating internationally;
changes in laws and regulations applicable to the Company's
business, including any pricing reform, and failure to comply with
such laws and regulations; inability to repay the Company's
existing indebtedness and uncertainties with respect to the
Company's ability to access future capital; and delays in the
execution of plans to build out an additional third-party
manufacturing facility approved by the appropriate regulatory
authorities and unexpected expenses associated with those
plans.
The Company may not actually achieve the results, plans,
intentions or expectations indicated by the Company's
forward-looking statements because, by their nature,
forward-looking statements involve risks and uncertainties because
they relate to events and depend on circumstances that may or may
not occur in the future. For additional information about the risks
and uncertainties that may affect the Company's business, please
see the factors discussed in Item 1A, "Risk Factors," in the
Company's Annual Report on Form 10-K for the year
ended December 31, 2020 and any subsequent Company filings
with the Securities and Exchange Commission (SEC).
The Company cautions readers not to place undue reliance on any
such forward-looking statements, which speak only as of the date of
this press release. The Company disclaims any obligation, except as
specifically required by law and the rules of the SEC, to publicly
update or revise any such statements to reflect any change in
expectations or in events, conditions or circumstances on which any
such statements may be based, or that may affect the likelihood
that actual results will differ from those set forth in the
forward-looking statements.
Contact:
Investors:
Eleanor Barisser
Associate Director, Investor Relations
Insmed
(718) 594-5332
eleanor.barisser@insmed.com
Media:
Mandy Fahey
Senior Director, Corporate Communications
Insmed
(732) 718-3621
amanda.fahey@insmed.com
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