BRIDGEWATER, N.J., Oct. 28, 2020 /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 the European Commission (EC) has granted
marketing authorization for ARIKAYCE® Liposomal 590 mg
Nebuliser Dispersion ("ARIKAYCE") for the treatment of
nontuberculous mycobacterial (NTM) lung infections caused by
Mycobacterium avium complex (MAC) in adults with limited
treatment options who do not have cystic fibrosis. Consideration
should be given to official guidance on the appropriate use of
antibacterial agents.
"We are thrilled that for the first time, non-CF patients with
NTM lung infections caused by MAC in the European Union (EU) have
an approved therapy to help manage this difficult-to-treat
condition, providing a new approach for those who have suffered
with few, or no, treatment options," said Will Lewis, Chair and Chief Executive Officer of
Insmed. "Today's approval underscores our commitment to serving the
MAC lung disease community around the world, and we look forward to
bringing ARIKAYCE to appropriate patients in the EU."
The EC approval of ARIKAYCE is based on results from the
randomized, open-label, global Phase 3 CONVERT study, which
demonstrated that once-daily ARIKAYCE, when combined with
multi-drug regimen (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.
"Today's approval marks a significant milestone in advancing
care for patients with MAC lung disease in the EU," said Professor
Michael Loebinger, Respiratory
Consultant at Royal Brompton Hospital, London, Professor of Practice (Respiratory
Medicine) at Imperial College, London, and an investigator in the CONVERT
study. "Currently, many patients fail to respond to the standard
treatment regimen and continue to suffer from the debilitating
effects of this rare and serious disease. Results from the landmark
CONVERT study show that adding ARIKAYCE has the potential to help
patients who were refractory to standard treatment achieve culture
conversion—a critical outcome."
The EC approval follows a positive opinion from the Committee
for Medicinal Products for Human Use of the European Medicines
Agency (EMA) on July 24, 2020. In
addition, the Committee for Orphan Medicinal Products of the EMA
has adopted a positive opinion recommending that ARIKAYCE maintain
Orphan Drug Designation in the EU for the treatment of NTM lung
disease, which was originally granted to Insmed in 2014.
Insmed plans to launch ARIKAYCE first in Germany, followed
by the United Kingdom (UK) and
other EU markets, subject to local reimbursement processes. As part
of Insmed's comprehensive approach to patient support, the Company
has established country-specific programs to provide patients with
direct and ongoing support and information.
"The treatment of MAC lung disease is challenging, with a
significant need for new therapies that improve upon the current
standard of care and offer options to patients who previously have
not been treated successfully," noted Marc
Lipman, Professor of Respiratory Medicine at University
College, London, and one of the
founding Trustees of NTM Patient Care UK.
In the United States, ARIKAYCE
(under the generic name amikacin liposome inhalation suspension),
is the first and only approved treatment for MAC lung disease as
part of a combination antibacterial drug regimen for adult patients
with limited or no alternative treatment options. Insmed has
submitted a new drug application for ARIKAYCE in Japan for the treatment of patients with NTM
lung disease caused by MAC who did not sufficiently respond to
prior treatment.
About MAC Lung Disease
Mycobacterium avium complex (MAC) lung disease is a
rare 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) and in the EU as ARIKAYCE® Liposomal 590 mg
Nebuliser Dispersion. 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 and new pharmaceutical formulations
that work together 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.
|
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,
ARIKAYCE® (amikacin liposome inhalation
suspension), is the first and only therapy approved in the
United States for the treatment of
refractory Mycobacterium avium complex (MAC) lung
disease as part of a combination antibacterial drug regimen for
adult patients with limited or no alternative treatment options.
MAC lung disease is a chronic, debilitating condition that can
cause severe and permanent lung damage. Insmed's earlier-stage
clinical pipeline includes brensocatib, a novel oral reversible
inhibitor of dipeptidyl peptidase 1 with therapeutic potential in
non-cystic fibrosis bronchiectasis and other inflammatory diseases,
and treprostinil palmitil, an inhaled formulation of a treprostinil
prodrug that may offer a differentiated product profile for rare
pulmonary disorders, including pulmonary arterial hypertension. 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. and the European Union or
for the Company's product candidates in the
U.S., Europe, Japan or other markets, including
the United Kingdom as a result of its recent exit from
the European Union; failure to successfully commercialize or
maintain U.S. approval for ARIKAYCE, the Company's only approved
product; 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; 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 FDA, including the risk that the Company will not
timely and successfully complete the study to validate a PRO tool
and complete the confirmatory post-marketing study required for
full approval of ARIKAYCE; inability of the Company, 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;
inaccuracies in the Company's estimates of the size of the
potential markets for ARIKAYCE 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; failure to obtain regulatory approval to
expand ARIKAYCE's indication to a broader patient population;
failure to successfully conduct future clinical trials for
ARIKAYCE; 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 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 limited experience operating
internationally; and changes in laws and regulations applicable to
the Company's business, including any pricing reform, and failure
to comply with such laws and regulations.
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, 2019, the Company's Quarterly Report on
Form 10-Q for the quarter ended June 30, 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:
Argot Partners
Laura Perry or Heather Savelle
(212) 600-1902
Insmed@argotpartners.com
Media:
Mandy Fahey
Senior Director, Corporate Communications
Insmed
(732) 718-3621
amanda.fahey@insmed.com
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