Insmed Announces that FDA Does Not Currently Plan to Hold Advisory Committee Meeting to Discuss New Drug Application for Brensocatib in Patients with Bronchiectasis

Rhea-AI Summary

Insmed (INSM) announced that the FDA does not currently plan to hold an advisory committee meeting to discuss the New Drug Application (NDA) for brensocatib in patients with non-cystic fibrosis bronchiectasis. The FDA has maintained the application under Priority Review with a PDUFA target action date of August 12, 2025.

The NDA is supported by data from the ASPEN study, the largest Phase 3 study ever conducted in bronchiectasis patients. If approved, brensocatib would become the first approved treatment for bronchiectasis and the first in a new class of medicines called dipeptidyl peptidase 1 (DPP1) inhibitors for treating neutrophil-mediated diseases.

Positive

• FDA granted Priority Review status for brensocatib NDA
• No advisory committee meeting requirement, potentially streamlining approval process
• Potential to be first-ever approved treatment for bronchiectasis
• First-in-class DPP1 inhibitor medicine

Negative

• None.

—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

FAQ

When is the PDUFA date for Insmed's brensocatib (INSM)?

The FDA has set a PDUFA target action date of August 12, 2025, for Insmed's brensocatib NDA.

What is the significance of FDA not requiring an advisory committee meeting for INSM's brensocatib?

This decision could indicate a more straightforward review process and potentially suggests that the FDA doesn't see any significant concerns that would require additional expert input.

What market opportunity does brensocatib represent for INSM?

Brensocatib has the potential to become the first and only approved treatment for bronchiectasis, representing a significant first-mover advantage in this market.

What clinical trial supports INSM's brensocatib NDA submission?

The NDA is supported by data from the ASPEN study, which was the largest Phase 3 study ever conducted in patients with bronchiectasis.

What type of FDA review status has INSM's brensocatib received?

Brensocatib has received Priority Review status from the FDA for its New Drug Application.