—FDA Reaffirms Application is Under Priority
Review with a PDUFA Target Action Date of August 12, 2025—
BRIDGEWATER, N.J., Feb. 24,
2025 /PRNewswire/ -- Insmed Incorporated (Nasdaq:
INSM), a people-first global biopharmaceutical company striving to
deliver first- and best-in-class therapies to transform the lives
of patients facing serious diseases, today announced that the U.S.
Food and Drug Administration (FDA) has informed the Company that it
does not currently plan to hold an advisory committee meeting to
discuss the New Drug Application (NDA) for brensocatib for patients
with non-cystic fibrosis bronchiectasis. The FDA provided this
update in its Day 74 communication to Insmed.
"We are very pleased with our ongoing communications with the
FDA about the NDA for brensocatib," said Martina Flammer, M.D., MBA, Chief Medical
Officer of Insmed. "We are committed to working closely with the
agency to successfully complete the review and potentially bring
forward this much-needed treatment for patients with
bronchiectasis."
The FDA had previously granted Priority Review to
Insmed's NDA for brensocatib and set a target action date
of August 12, 2025, under the Prescription Drug User Fee Act
(PDUFA). This NDA is based on data from the landmark ASPEN study, the largest Phase 3 study ever
conducted in patients with bronchiectasis. Brensocatib has the
potential to become the first and only approved treatment for
bronchiectasis and the first in a new class of medicines called
dipeptidyl peptidase 1 (DPP1) inhibitors for the treatment of
neutrophil-mediated diseases.
About Bronchiectasis
Bronchiectasis is a serious, chronic lung disease in which the
bronchi become permanently dilated due to a cycle of infection,
inflammation, and lung tissue damage. The condition is marked by
frequent pulmonary exacerbations requiring antibiotic therapy
and/or hospitalizations. Symptoms include chronic cough, excessive
sputum production, shortness of breath, and repeated respiratory
infections, which can worsen the underlying condition. Today,
approximately 500,000 patients in the U.S., 600,000 patients in the
EU5 (France, Germany, Italy, Spain,
and UK), and 150,000 patients in Japan have been diagnosed with bronchiectasis,
and there are currently no approved therapies specifically
targeting bronchiectasis in these regions.
About Brensocatib
Brensocatib is a small molecule, oral, reversible inhibitor of
dipeptidyl peptidase 1 (DPP1) being developed by Insmed for the
treatment of patients with bronchiectasis, chronic rhinosinusitis
without nasal polyps, hidradenitis suppurativa, and other
neutrophil-mediated diseases. DPP1 is an enzyme responsible for
activating neutrophil serine proteases (NSPs), such as neutrophil
elastase, in neutrophils when they are formed in the bone marrow.
Neutrophils are the most common type of white blood cell and play
an essential role in pathogen destruction and inflammatory
mediation. In chronic inflammatory lung diseases, neutrophils
accumulate in the airways and result in excessive active NSPs that
cause lung destruction and inflammation. Brensocatib may decrease
the damaging effects of inflammatory diseases such as
bronchiectasis by inhibiting DPP1 and its activation of NSPs.
Brensocatib is an investigational drug product that has not been
approved for any indication in any jurisdiction.
About Insmed
Insmed Incorporated is a people-first global biopharmaceutical
company striving to deliver first- and best-in-class therapies to
transform the lives of patients facing serious diseases. The
Company is advancing a diverse portfolio of approved and mid- to
late-stage investigational medicines as well as cutting-edge drug
discovery focused on serving patient communities where the need is
greatest. Insmed's most advanced programs are in pulmonary and
inflammatory conditions, including a therapy approved in
the United States, Europe, and Japan to treat a chronic, debilitating lung
disease. The Company's pre-clinical research programs encompass a
wide range of technologies and modalities, including gene therapy,
AI-driven protein engineering, protein manufacturing, RNA
end-joining, and synthetic rescue.
Headquartered in Bridgewater, New
Jersey, Insmed has offices and research locations throughout
the United States, Europe, and Japan. Insmed is proud to be recognized as one
of the best employers in the biopharmaceutical industry, including
spending four consecutive years as the No. 1 Science Top
Employer. Visit www.insmed.com to learn more.
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 timings discussed, projected,
anticipated or indicated in any forward-looking statements. Such
risks, uncertainties and other factors include, among others, the
following: the risk that the full data set from the ASPEN study or data generated in further
clinical trials of brensocatib will not be consistent with the
topline results of the ASPEN study
or any additional results of the ASPEN study; failure to obtain, or delays in
obtaining, regulatory approvals for brensocatib in the U.S.,
Europe or Japan; failure to successfully commercialize
brensocatib, if approved by applicable regulatory authorities, in
the U.S., Europe or Japan, or to maintain U.S., European or
Japanese approval for brensocatib once approved; uncertainties in
the degree of market acceptance of brensocatib by physicians,
patients, third-party payors and others in the healthcare
community; inaccuracies in the Company's estimates of the size of
the potential markets for brensocatib 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; inability of the Company, Esteve Química,
S.A., Thermo Fisher Scientific, Inc. or the Company's other
third-party manufacturers to comply with regulatory requirements
related to brensocatib; the Company's inability to obtain adequate
reimbursement from government or third-party payors for brensocatib
or acceptable prices for brensocatib; development of unexpected
safety or efficacy concerns related to brensocatib; failure to
obtain regulatory approval for potential future brensocatib
indications; restrictions or other obligations imposed on us by
agreements related to brensocatib, including our license agreement
with AstraZeneca AB, and failure to comply with our obligations
under such agreements; failure to successfully conduct future
clinical trials for brensocatib, including due to the Company's
potential inability to enroll or retain sufficient patients to
conduct and complete the trials or generate data necessary for
regulatory approval, among other things; risks that the Company's
clinical studies will be delayed or that serious side effects will
be identified during drug development; failure of third parties on
which the Company is dependent to manufacture sufficient quantities
of brensocatib for commercial or clinical needs, to conduct the
Company's clinical trials, or to comply with the Company's
agreements or laws and regulations that impact the Company's
business or agreements with the Company; the strength and
enforceability of the Company's intellectual property rights or the
rights of third parties; the cost and potential reputational damage
resulting from litigation to which the Company may become a party,
including product liability claims; changes in laws and regulations
applicable to the Company's business and failure to comply with
such laws and regulations; business or economic disruptions due to
catastrophes or other events, including natural disasters or public
health crises; and inability to repay the Company's existing
indebtedness and uncertainties with respect to the Company's need
and ability to access future capital.
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, 2024 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:
Bryan Dunn
Vice President, Investor Relations
(646) 812-4030
bryan.dunn@insmed.com
Michael V. Morabito, Ph.D.
Director, Investor Relations
(917) 936-8430
michael.morabito@insmed.com
Gianna De Palma
Manager, Investor Relations
(973) 886-2236
gianna.depalma@insmed.com
Media:
Mandy Fahey
Vice President, Corporate Communications
(732) 718-3621
amanda.fahey@insmed.com
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SOURCE Insmed Incorporated
