Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical
company focused on the unmet needs of patients with rare diseases,
today announced that it has withdrawn its Marketing Authorization
Application (MAA) from the European Medicines Agency (EMA) for
ARIKAYCE™ for the treatment of nontuberculous mycobacteria (NTM)
lung disease.
The MAA filing was based on data from the company’s
completed phase 2 study. During the May 2016 Committee for
Medicinal Products for Human Use (CHMP) meeting, the CHMP indicated
that the phase 2 study did not provide a sufficient amount of
evidence to support an approval. Insmed intends to resubmit its MAA
when clinical data from its ongoing global phase 3 study are
available.
“We remain on track to complete patient enrollment
later this year in our landmark global phase 3 CONVERT study,” said
Will Lewis, president and chief executive officer of Insmed.
“ARIKAYCE has the potential to make a significant difference to
patients whose NTM lung disease persists despite long-term courses
of multi-drug regimens. We are committed to achieving our ultimate
goal of making ARIKAYCE available to patients in the US, Europe,
and Asia who are living with the devastating effects of this
disease.”
The company will continue to support numerous
compassionate use programs in Europe for certain patients whose
physicians believe they may benefit from ARIKAYCE but are unable to
participate in the clinical study. Compassionate use programs
are generally intended to make products available on a
named-patient basis before they are approved or commercially
available in accordance with local regulations. The company is
currently supporting the compassionate use of ARIKAYCE in France,
Germany, and the Netherlands. The company also expects to
support a compassionate use program in Italy.
About Insmed
Insmed Incorporated is a global biopharmaceutical
company focused on the unmet needs of patients with rare diseases.
The company is advancing a global phase 3 clinical study of
ARIKAYCE (liposomal amikacin for inhalation) in nontuberculous
mycobacteria (NTM) lung disease, a rare and often chronic infection
that is capable of causing irreversible lung damage and can be
fatal. There are currently no products indicated for the treatment
of NTM lung disease in the United States or European Union (EU).
Insmed’s earlier-stage clinical pipeline includes INS1009, a
nebulized prodrug formulation of treprostinil that the company
believes may offer a differentiated product profile with
therapeutic potential in rare pulmonary disorders such as pulmonary
arterial hypertension (PAH), idiopathic pulmonary fibrosis (IPF)
sarcoidosis, and severe refractory asthma. To complement its
internal research, Insmed actively seeks in-licensing opportunities
for a broad range of rare diseases. For more information,
visit www.insmed.com.
“Insmed” and “ARIKAYCE” are the company's
trademarks. All other trademarks, trade names or service marks
appearing in this press release are the property of their
respective owners.
About Nontuberculous Mycobacteria Lung
Disease
NTM is a rare and serious disorder associated with
increased morbidity and mortality. There is an increasing rate of
lung disease caused by NTM and this 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. There are no products specifically
indicated for the treatment of NTM lung disease in the US, Europe
and Canada. Current guideline-based approaches involve multi-drug
regimens that may cause severe side effects 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 in the
US is increasing at approximately 8% per year and that NTM patients
on Medicare over the age of 65 are 40% more likely to die over the
period of the study than those who did not have the disease. A 2015
publication from co-authors from several US government departments
projected 181,037 national annual cases in 2014 costing the US
healthcare system approximately $1.7 billion.
For more information about NTM lung disease, visit
NTMfacts.com.
About ARIKAYCE
ARIKAYCE, or liposomal amikacin for inhalation, is
a novel, once daily formulation of amikacin that is in late stage
clinical development for patients with NTM lung disease. Amikacin
solution for parenteral administration is an established drug that
is effective 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.
ARIKAYCE’s ability to deliver high levels of amikacin directly to
the lung distinguishes it from intravenous amikacin. ARIKAYCE is
administered once daily using an optimized, investigational eFlow®
Nebulizer System manufactured by PARI Pharma GmbH, a novel, highly
efficient and portable aerosol delivery system.
Forward-looking statements
This press release contains forward looking statements.
“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)
identify forward-looking statements.
Forward-looking statements are based upon the company’s current
expectations and beliefs, and involve known and unknown risks,
uncertainties and other factors, which may cause 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 factors include, among others, the
factors discussed in Item 1A “Risk Factors” in the company’s Annual
Report on Form 10-K for the year ended December 31, 2015 and
subsequent quarterly reports on Form 10-Q, and the following: the
ability to complete development of, receive regulatory approval
for, and successfully commercialize ARIKAYCE, or liposomal amikacin
for inhalation (LAI), and INS1009, nebulized treprostinil prodrug;
estimates of expenses and future revenues and profitability; plans
to develop and market new products and the timing of these
development programs; status, timing, and the results of
preclinical studies and clinical trials and preclinical and
clinical data described herein; the timing of responses to
information and data requests from the US Food and Drug
Administration, the European Medicines Agency, and other regulatory
authorities; clinical development of product candidates; ability to
obtain and maintain regulatory approval for product candidates;
expectation as to the timing of regulatory review and approval;
estimates regarding our capital requirements and the needs for
additional financing; estimates of the size of the potential
markets for product candidates; selection and licensing of product
candidates; ability to attract third parties with acceptable
development, regulatory and commercialization expertise; the
benefits to be derived from corporate license agreements and other
third party efforts, including those relating to the development
and commercialization of product candidates; the degree of
protection afforded to the company by its intellectual property
portfolio; the safety and efficacy of product candidates; sources
of revenues and anticipated revenues, including contributions from
license agreements and other third party efforts for the
development and commercialization of products; ability to create an
effective direct sales and marketing infrastructure for products
the company elects to market and sell directly; the rate and degree
of market acceptance of product candidates; the timing, scope and
rate of reimbursement for product candidates; the success of other
competing therapies that may become available; and the availability
of adequate supply and manufacturing capacity and quality for
product candidates.
The company cautions readers not to place undue reliance on any
such forward-looking statements, which speak only as of the date
they are made. Insmed 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.
Insmed Incorporated:
Susan Mesco
Head of Investor Relations
908-947-4326
Insmed (NASDAQ:INSM)
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