—Commercial availability expected in early Q4
2018—
Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical
company focused on the unmet needs of patients with rare diseases,
today announced that the U.S. Food and Drug Administration (FDA)
has granted accelerated approval of ARIKAYCE® (amikacin liposome
inhalation suspension) for the treatment of Mycobacterium avium
complex (MAC) lung disease as part of a combination antibacterial
drug regimen for adult patients who have limited or no alternative
treatment options. ARIKAYCE is the first and only therapy approved
in the U.S. specifically for patients with MAC lung disease, a
chronic and debilitating condition that can significantly increase
patient morbidity and mortality.
ARIKAYCE is the first product approved via the Limited
Population Pathway for Antibacterial and Antifungal Drugs (LPAD).
LPAD, which was enacted as part of the 21st Century Cures Act,
serves to advance the development of new antibacterial drugs to
treat serious or life-threatening infections in limited populations
of patients with unmet needs.
“Today’s approval is a momentous occasion for all of us living
with and advocating for people with MAC lung disease,” said Philip
Leitman, President of Nontuberculous Mycobacteria Info &
Research (NTMir). “ARIKAYCE provides a much-needed treatment for
patients with this chronic and life-threatening disease who have
not responded to the current standard of care. Many of these
patients have been suffering for years and face significant
challenges in their day-to-day lives, and we are excited to finally
have an approved treatment for them.”
“Patients suffering from MAC lung disease who have not responded
to available guideline-based therapies face a restricted quality of
life due to this debilitating and progressive illness. As a
physician and Principal Investigator in the CONVERT study, I am
extremely pleased that there is now a therapy specifically studied
in and approved for patients with MAC lung disease who currently
have limited or no treatment options,” said David Griffith, M.D.,
Professor of Medicine, W.A. and E.B. Moncrief Distinguished
Professor at The University of Texas Health Science Center.
“ARIKAYCE has the potential to address the significant unmet needs
in this difficult-to-treat population.”
Physicians can begin prescribing ARIKAYCE immediately and Insmed
expects product to be available in select specialty pharmacies in
the coming weeks. Insmed is committed to providing access to
ARIKAYCE for appropriate patients with MAC lung disease and to
supporting these patients throughout their treatment journey. The
Company has launched the Arikares Support Program, which provides
dedicated coordinators to help patients navigate the reimbursement
process and trainers who can familiarize patients with how to use
ARIKAYCE. Patients prescribed ARIKAYCE can call 1-833-ARIKARE to
enroll in the support program.
“The approval of ARIKAYCE is a significant moment for adult
patients suffering from MAC lung disease who have limited or no
available treatment options. It also represents an incredible
milestone for our company, which has taken this medicine from
concept to approval and now will launch the drug across the U.S.
Our mission is to address the unmet needs of patients with serious
and rare diseases, and we are thrilled to be able to provide the
first-ever approved therapy specifically for patients in the U.S.
with MAC lung disease,” said Will Lewis, President and Chief
Executive Officer of Insmed. “I want to thank the patients and
physicians who have made this milestone possible through their
participation in the clinical trials, as well as our dedicated
Insmed team. We look forward to focusing our efforts on the launch
of ARIKAYCE in the U.S.”
The approval of ARIKAYCE under FDA’s LPAD and accelerated
approval pathways was based on results from the ongoing Phase 3
CONVERT study, which has demonstrated that ARIKAYCE, when combined
with guideline-based therapy (GBT), improved sputum culture
conversion rates. The global CONVERT study met its primary endpoint
of sputum culture conversion by Month 6 with statistical
significance for once-daily ARIKAYCE when added to GBT compared
with GBT alone (p<0.0001) in patients with refractory
nontuberculous mycobacterial (NTM) lung disease caused by MAC. In
the study, the addition of ARIKAYCE to GBT eliminated evidence of
NTM lung disease caused by MAC in sputum by Month 6 in 29% of
patients, compared to 9% of patients on GBT alone.
Patients who are prescribed ARIKAYCE will be provided with a
Medication Guide containing important safety information set forth
below, including the boxed warning, as well as full Instructions
for Use and a step-by-step guide to using the product. Insmed
also will send health care providers a letter describing the scope
of the limited population approval and the potential risk of
respiratory adverse reactions.
As a condition of accelerated approval, Insmed is collaborating
with the FDA on the design of an additional clinical study to
support full approval. The study design is currently under
discussion with FDA and is proposed to be a randomized,
double-blind, placebo-controlled clinical trial to assess and
describe the clinical benefit of ARIKAYCE in patients with NTM lung
disease caused by MAC. The trial will evaluate the effect of
ARIKAYCE on a clinically meaningful endpoint, as compared to an
appropriate control, in the intended patient population of patients
with MAC infection. Insmed will provide additional updates once the
study design has been finalized with FDA. Continued approval of
ARIKAYCE will be contingent upon verification and description of
clinical benefit in this study.
ARIKAYCE is administered once daily using the Lamira™ Nebulizer
System (PARI Pharma GmbH [PARI]).
Conference Call Information
Insmed will host a conference call today at 6:15 PM Eastern Time
to discuss the FDA approval. The call can be accessed by dialing
(844) 707-0669 (U.S. and Canada) or (703) 639-1223 (international)
and entering the conference ID number 2376004. The call will also
be webcast live on the Company's website at www.insmed.com.
A replay of the conference call will be accessible approximately
two hours after its completion through October 5, 2018 by dialing
(855) 859-2056 (domestic) or (404) 537-3406 (international) and
referencing conference ID number 2376004. A webcast of the call
will also be archived for 90 days under the Investor Relations
section of the Company's website at www.insmed.com.
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 needs. Current guideline-based treatment
involves the use of multi-drug regimens that are not specifically
approved for MAC lung disease. The course of treatment is often two
years or more and is inadequate in treating the disease in many
patients.
About ARIKAYCE® (amikacin liposome
inhalation suspension)
ARIKAYCE is the first and only FDA-approved therapy indicated
for the treatment of Mycobacterium avium complex (MAC) lung disease
as part of a combination antibacterial drug regimen for adult
patients with limited or no alternative treatment options. 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 it is taken up by lung macrophages where the
infection resides. This approach prolongs the release of amikacin
in the lungs while limiting systemic exposure. ARIKAYCE is
administered once daily using the Lamira™ Nebulizer System
manufactured by PARI Pharma GmbH.
About PARI Pharma and the
Lamira™ Nebulizer
System
ARIKAYCE® (amikacin liposome inhalation suspension) 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 liquid medications, including
liposomal formulations such as 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.
About CONVERT (INS-212) and INS-312
CONVERT is a randomized, open-label, global Phase 3 trial
designed to confirm the sputum culture conversion results seen in
Insmed's Phase 2 clinical trial of ARIKAYCE in patients with
refractory NTM lung disease caused by MAC. CONVERT is being
conducted in 18 countries at more than 125 sites. The primary
efficacy endpoint is the proportion of patients who achieved sputum
culture conversion at Month 6 in the ARIKAYCE plus GBT arm compared
to the GBT-only arm. Patients who achieved sputum culture
conversion by Month 6 are continuing in the CONVERT study for an
additional 12 months of treatment following the first monthly
negative sputum culture. Patients who did not culture convert may
have been eligible to enroll in our INS-312 study. INS-312 is a
single-arm open-label extension study for patients who completed
six months of treatment in the INS-212 study but did not
demonstrate culture conversion by Month 6. Under the study
protocol, non-converting patients in the ARIKAYCE plus GBT arm
of the INS-212 study will receive an additional 12 months of
ARIKAYCE plus GBT. Patients who crossed over from the GBT-only arm
of the INS-212 study will receive 12 months of treatment of
ARIKAYCE plus GBT.
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 ARIKAYCE® (amikacin
liposome inhalation suspension), which is approved in the United
States for the treatment of 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 rare and often chronic infection that can
cause irreversible lung damage and can be fatal. Insmed's
earlier-stage clinical pipeline includes INS1007, a novel oral
reversible inhibitor of dipeptidyl peptidase 1 with therapeutic
potential in non-cystic fibrosis bronchiectasis and other
inflammatory diseases, and INS1009, an inhaled nanoparticle
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.
IMPORTANT SAFETY INFORMATION
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.
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.
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 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.
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: risks that the remainder of the data from the treatment
and off-treatment phases of INS-212 will not be consistent with the
six-month results of the study; uncertainties in the research and
development of the Company’s existing product candidates, including
due to delays in data readouts, such as the full data from the
INS-212 study, patient enrollment and retention or failure of the
Company’s preclinical studies or clinical trials to satisfy
pre-established endpoints, including secondary endpoints in the
INS-212 study and endpoints in the INS-212 extension study (the
INS-312 study); risks that subsequent data from the INS-312 study
will not be consistent with the interim results; imposition of
significant post-approval regulatory requirements on our product
candidates, including a requirement for a post-approval
confirmatory clinical study, or failure to maintain or obtain full
regulatory approval for the Company’s product candidates, if
received, due to a failure to satisfy post-approval regulatory
requirements, such as the submission of sufficient data from a
confirmatory clinical study; safety and efficacy concerns related
to the Company’s product candidates; uncertainties in the rate and
degree of market acceptance of product candidates, if approved;
inability to create an effective direct sales and marketing
infrastructure or to partner with third parties that offer such an
infrastructure for distribution of the Company’s product
candidates, if approved; failure to obtain, or delays in obtaining,
regulatory approval from the U.S. Food and Drug Administration,
Japan’s Ministry of Health, Labour and Welfare, Japan’s
Pharmaceuticals and Medical Devices Agency, the European Medicines
Agency, and other regulatory authorities for the Company’s product
candidates or their delivery devices, including due to insufficient
clinical data, selection of endpoints that are not satisfactory to
regulators or complexity in the review process for combination
products; lack of experience in conducting and managing preclinical
development activities and clinical trials necessary for regulatory
approval, including the regulatory filing and review process;
inaccuracies in the Company’s estimates of the size of the
potential markets for the Company’s product candidates or
limitations by regulators on the proposed treatment population for
the Company’s product candidates; failure of third parties on which
the Company is dependent to conduct the Company’s clinical trials,
to manufacture sufficient quantities of the Company’s product
candidates for clinical or commercial needs, including the
Company’s raw materials suppliers, or to comply with the Company’s
agreements or laws and regulations that impact the Company’s
business; inaccurate estimates regarding the Company’s future
capital requirements, including those necessary to fund the
Company’s ongoing clinical development, regulatory and
commercialization efforts as well as milestone payments or
royalties owed to third parties; failure to develop, or to license
for development, additional product candidates, including a failure
to attract experienced third-party collaborators; uncertainties in
the timing, scope and rate of reimbursement for the Company’s
product candidates; changes in laws and regulations applicable to
the Company’s business and failure to comply with such laws and
regulations; inability to repay the Company’s existing indebtedness
or to obtain additional capital when needed on desirable terms or
at all; failure to obtain, protect and enforce the Company’s
patents and other intellectual property and costs associated with
litigation or other proceedings related to such matters;
restrictions imposed on the Company by license agreements that are
critical for the Company’s product development, including the
Company’s license agreements with PARI Pharma GmbH and AstraZeneca
AB, and failure to comply with the Company’s obligations under such
agreements; competitive developments affecting the Company’s
product candidates and potential exclusivity related thereto; the
cost and potential reputational damage resulting from litigation to
which the Company is or may become a party; loss of key personnel;
and lack of experience operating internationally.
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, 2017 and any subsequent Company filings with the Securities and
Exchange Commission.
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 Securities and
Exchange Commission, 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:
Blaine Davis Insmed Incorporated (908)
947-2841blaine.davis@insmed.com
Insmed (NASDAQ:INSM)
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