Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical
company focused on the unmet needs of patients with rare diseases,
announced today that the U.S. Food and Drug
Administration (FDA) has notified the company that the New
Drug Application (NDA) for ALIS (Amikacin Liposome Inhalation
Suspension) for adult patients with nontuberculous mycobacterial
(NTM) lung disease caused by Mycobacterium avium complex
(MAC) has been accepted and will be considered filed on May 27,
2018. The FDA has completed the filing review and determined
that the application is sufficiently complete to permit a
substantive review. The FDA granted Insmed’s
request for Priority Review and has set an action date
of September 28, 2018 under the Prescription Drug User
Fee Act (PDUFA).
“We are pleased that the FDA has accepted our NDA with Priority
Review, which underscores the urgent need for therapies which
address NTM lung disease caused by MAC,” said Will Lewis, President
and Chief Executive Officer of Insmed. “ALIS has the potential to
be the first approved inhaled therapy for the treatment of this
serious and debilitating lung infection in the United States, and
we look forward to working with the Agency through the review
process.”
Insmed continues to expect that the FDA will convene an advisory
committee meeting to discuss this application.
Priority Review applies to an application for a drug that treats
a serious condition and that, if approved, would provide a
significant improvement in safety or effectiveness. FDA has
previously designated ALIS as an orphan drug, a breakthrough
therapy and a Qualified Infectious Disease Product (QIDP) under the
Generating Antibiotic Incentives Now (GAIN) Act.
About NTM Lung Disease
NTM lung disease is a rare and serious disorder associated with
increased rates of morbidity and mortality. There is an increasing
prevalence of lung disease caused by NTM, and Insmed believes it is
an emerging public health concern worldwide. Patients with NTM lung
disease may experience a multitude of symptoms such as fever,
weight loss, cough, lack of appetite, night sweats, blood in the
sputum, and fatigue. Patients with NTM lung disease frequently
require lengthy hospital stays to manage their condition. Insmed is
not aware of any approved inhaled therapies specifically indicated
for refractory NTM lung disease caused by MAC in North America,
Japan or Europe. Current guideline-based approaches involve use of
multi-drug regimens not approved for the treatment of NTM lung
disease, and treatment can be as long as two years or more.
The prevalence of human disease attributable to NTM has
increased over the past two decades. In a decade long study (1997
to 2007), researchers found that the prevalence of NTM lung disease
in the U.S. was increasing at approximately 8% per year and that
NTM patients on Medicare over the age of 65 were 40% more likely to
die over the period of the study than those who did not have the
disease. In the U.S., Insmed estimates there will be between 75,000
and 105,000 patients with diagnosed NTM lung disease in 2018, of
which the Company expects 40,000 to 50,000 will be treated for NTM
lung disease caused by MAC. Insmed expects that between 10,000 and
15,000 of these patients will be refractory to treatment. In Japan,
Insmed estimates there will be between 125,000 and 145,000 patients
with diagnosed NTM lung disease in 2018, with approximately 60,000
to 70,000 of those patients being treated for NTM lung disease
caused by MAC and 15,000 to 18,000 of these treated patients being
refractory to treatment. Insmed also estimates there will be
approximately 14,000 patients with diagnosed NTM lung disease in
the EU5 (comprised of France, Germany, Italy, Spain and the United
Kingdom) in 2018, of which the Company estimates approximately
4,400 will be treated for NTM lung disease caused by MAC and
approximately 1,400 of these treated patients will be refractory to
treatment.
About ALIS
ALIS is a novel, inhaled, once-daily formulation of amikacin
that is in late-stage clinical development for adult patients with
treatment-refractory NTM lung disease caused by MAC. Amikacin
solution for parenteral administration is an established drug that
has activity against a variety of NTM; however, its use is limited
by the need to administer it intravenously and by toxicity to
hearing, balance, and kidney function. Insmed's advanced pulmonary
liposome technology uses charge neutral liposomes to deliver
amikacin directly to the lung where it is taken up by the lung
macrophages where the NTM infection resides. This prolongs the
release of amikacin in the lungs while minimizing systemic exposure
thereby offering the potential for decreased systemic toxicities.
ALIS’s ability to deliver high levels of amikacin directly to the
lung distinguishes it from intravenous amikacin. ALIS is
administered once daily using an optimized, investigational eFlow®
Nebulizer System manufactured by PARI Pharma GmbH (PARI), a
portable aerosol delivery system.
About Insmed
Insmed Incorporated is a global biopharmaceutical company
focused on the unmet needs of patients with rare diseases. The
Company’s lead product candidate is ALIS, which is in late-state
development for adult patients with treatment refractory NTM lung
disease caused by MAC, which is a rare and often chronic infection
that is capable of causing 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.
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 data from the remainder of the treatment
and off-treatment phases of INS-212 will not be consistent with the
top-line 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; 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, such as
the eFlow Nebulizer System, including due to insufficient clinical
data, selection of endpoints that are not satisfactory to
regulators, complexity in the review process for combination
products or inadequate or delayed data from a human factors study
required for U.S. regulatory approval; failure to maintain
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
confirmatory clinical studies; safety and efficacy concerns related
to the Company’s product candidates; lack of experience in
conducting and managing preclinical development activities and
clinical trials necessary for regulatory approval, including the
regulatory filing and review process; failure to comply with
extensive post-approval regulatory requirements or imposition of
significant post-approval restrictions on the Company’s product
candidates by regulators; 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;
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 be 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 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) 947-2841
blaine.davis@insmed.com
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