— ARIKAYCE is the First and Only Therapy Approved in
Both the European Union and United
States for This Difficult-To-Treat Condition —
— Planned Product Launches to Begin in Germany, Followed by UK and Other EU Countries
—
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.
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
Logo -
https://mma.prnewswire.com/media/801462/Insmed_Logo.jpg