CHMP Recommends EU Approval of BRINSUPRI™ (brensocatib) for the Treatment of Non-Cystic Fibrosis Bronchiectasis

— If Approved, BRINSUPRI Would Be the First and Only Treatment in the European Union for Non-Cystic Fibrosis Bronchiectasis (NCFB) and the First-in-Class DPPI Inhibitor Targeting Neutrophilic Inflammation

—   BRINSUPRI Was Reviewed Under CHMP's Accelerated Assessment Pathway as It Is Considered of Major Interest for Public Health and Therapeutic Innovation —

BRIDGEWATER, N.J., Oct. 17, 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 Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of its DPP1 inhibitor BRINSUPRI (brensocatib 25 mg tablets) for the treatment of non-cystic fibrosis bronchiectasis (NCFB) in patients 12 years of age and older with two or more exacerbations in the prior 12 months. If approved, BRINSUPRI would be first-in-class and the first approved therapy in the European Union to treat NCFB.

"Non-cystic fibrosis bronchiectasis is a progressive disease that can lead to lung function decline—and with diagnoses rising steadily worldwide, there is an urgent need for additional treatment options," said ASPEN lead study investigator James Chalmers, MBChB, Ph.D., Professor and Consultant Respiratory Physician at the School of Medicine, University of Dundee, UK. "Based on the clinical benefits demonstrated in the ASPEN and WILLOW studies, brensocatib has the potential to change the treatment paradigm in non-cystic fibrosis bronchiectasis, offering the prospect of significantly improving treatment outcomes for those living with this debilitating illness."

The CHMP opinion is based on a comprehensive scientific evaluation of the marketing authorization application, including data from the Phase 3 ASPEN and Phase 2 WILLOW studies, which were both published in the New England Journal of Medicine. BRINSUPRI was reviewed under accelerated assessment by the EMA as it is deemed to be of major interest for public health and therapeutic innovation. BRINSUPRI had previously been granted Priority Medicines (PRIME) designation by the EMA for the treatment of NCFB following positive Phase 2 study results from WILLOW. The European Commission (EC) will review the CHMP opinion, with a final decision anticipated in the coming months.

"With this positive CHMP opinion and the recent FDA approval of BRINSUPRI, we are witnessing the beginning of a new chapter for patients living with NCFB - a serious, chronic lung condition that has long lacked approved treatment options in the European Union," said Martina Flammer, M.D., MBA, Chief Medical Officer of Insmed. "For too long, patients and families have had to endure the daily burden of this disease without a treatment specifically developed to treat bronchiectasis. At Insmed, our focus remains on delivering first- and best-in-class therapies to patients with serious diseases, and this important milestone brings us one step closer to potentially bringing the first-ever approved treatment to NCFB patients in Europe."

About BRINSUPRI™ (brensocatib)

BRINSUPRI™ (brensocatib) is a small molecule, once-daily, oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1) and is indicated in the United States for the treatment of non-cystic fibrosis bronchiectasis (NCFB) in adult and pediatric patients 12 years of age or older. Brensocatib is designed to inhibit the activation of enzymes (neutrophil serine proteases) in neutrophils that are key drivers of chronic airway inflammation in NCFB. Brensocatib is also being evaluated for its potential role in other neutrophil-mediated diseases.

About ASPEN

ASPEN was a global, randomized, double-blind, placebo-controlled Phase 3 study to assess the efficacy, safety, and tolerability of brensocatib in patients with non-cystic fibrosis bronchiectasis (NCFB). As part of the ASPEN study's conduct, more than 460 trial sites were engaged in nearly 40 countries. After excluding sites that did not enroll any patients and all sites in Ukraine, the total number of active sites in ASPEN was 391 sites in 35 countries. Adult patients (ages 18 to 85 years) were randomized 1:1:1 and adolescent patients (ages 12 to <18 years) were randomized 2:2:1 for treatment with brensocatib 10 mg, brensocatib 25 mg, or placebo once daily for 52 weeks, followed by 4 weeks off treatment. The primary efficacy analysis included data from 1,680 adult patients and 41 adolescent patients.

About WILLOW

WILLOW was a randomized, double-blind, placebo-controlled, parallel-group, multi-center, multi-national, Phase 2 study to assess the efficacy, safety and tolerability, and pharmacokinetics of brensocatib administered once daily for 24 weeks in patients with non-cystic fibrosis bronchiectasis (NCFB). WILLOW was conducted at 116 sites and enrolled 256 adult patients diagnosed with NCFB who had at least two documented pulmonary exacerbations in the 12 months prior to screening. Patients were randomized 1:1:1 to receive either 10 mg or 25 mg of brensocatib or matching placebo. The primary efficacy endpoint was the time to first pulmonary exacerbation over the 24-week treatment period in the brensocatib arms compared to the placebo arm.

About Bronchiectasis  

NCFB is a serious, chronic, and progressive inflammatory lung disease in which the bronchi become permanently dilated due to a cycle of infection, inflammation, lung tissue damage and mucociliary dysfunction. The disease is marked by frequent pulmonary exacerbations requiring antibiotic therapy and/or hospitalizations. Symptoms include chronic cough, excessive sputum production, shortness of breath, fatigue and repeated respiratory infections, which can worsen the underlying disease. 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 NCFB. Outside the U.S. there are currently no approved therapies specifically targeting bronchiectasis in these regions.

BRINSUPRI™ (brensocatib) U.S. INDICATION AND IMPORTANT SAFETY INFORMATION

Indication in the U.S.

BRINSUPRI is indicated for the treatment of non-cystic fibrosis bronchiectasis (NCFB) in adult and pediatric patients 12 years of age and older.  

Important Safety Information in the U.S.

WARNINGS AND PRECAUTIONS

Dermatologic Adverse Reactions

Treatment with BRINSUPRI is associated with an increase in dermatologic adverse reactions, including rash, dry skin, and hyperkeratosis. Monitor patients for development of new rashes or skin conditions and refer patients to a dermatologist for evaluation of new dermatologic findings. 

Gingival and Periodontal Adverse Reactions

Treatment with BRINSUPRI is associated with an increase in gingival and periodontal adverse reactions. Refer patients to dental care services for regular dental checkups while taking BRINSUPRI. Advise patients to perform routine dental hygiene. 

Live Attenuated Vaccines

It is unknown whether administration of live attenuated vaccines during BRINSUPRI treatment will affect the safety or effectiveness of these vaccines. The use of live attenuated vaccines should be avoided in patients receiving BRINSUPRI. 

ADVERSE REACTIONS

The most common adverse reactions ≥2% in the ASPEN trial included upper respiratory tract infection, headache, rash, dry skin, hyperkeratosis, and hypertension. The safety profile for adult patients with NCFB in WILLOW was generally similar to ASPEN, except for a higher incidence of gingival and periodontal adverse reactions. 

Less Common Adverse Reactions 

Liver Function Test Elevations

In ASPEN, there was an increase from baseline in average ALT, AST, and alkaline phosphatase levels at all time points from Week 4 through Week 56 in both BRINSUPRI 10 mg and 25 mg arms compared to placebo. The incidence of ALT >3X upper limit of normal (ULN) was 0%, 1.2%, and 0.9%; the incidence of AST >3X ULN was 0.2%, 0.3%, and 0.5%; and the incidence of alkaline phosphatase >1.5X ULN was 2.5%, 4.1%, and 4.0% in patients treated with placebo and BRINSUPRI 10 mg and 25 mg, respectively. 

Skin Cancers

In ASPEN, the incidence of skin cancers among patients treated with BRINSUPRI 10 mg and 25 mg was 0.5% and 1.9%, respectively, compared to 1.1% in placebo-treated patients. 

Alopecia

In ASPEN, the incidence of alopecia among patients treated with BRINSUPRI 10 mg and 25 mg was 1.5% and 1.6% respectively, compared to 0.4% in placebo-treated patients.  

USE IN SPECIFIC POPULATIONS

Pregnancy: There are no clinical data on the use of BRINSUPRI in pregnant women. 

Lactation: There is no information regarding the presence of BRINSUPRI and/or its metabolite(s) in human milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for BRINSUPRI and any potential adverse effects on the breastfed child from BRINSUPRI or from the underlying maternal condition. 

Pediatric use: The safety and effectiveness of BRINSUPRI for the treatment of NCFB have been established in pediatric patients aged 12 years and older. Common adverse reactions in pediatric patients aged 12 years and older enrolled in ASPEN were consistent with those in adults. The safety and effectiveness of BRINSUPRI have not been established in pediatric patients younger than 12 years of age. 

Please see full US Prescribing Information. 

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 two approved therapies to treat chronic, debilitating lung diseases. The Company's early-stage 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 or follow us on LinkedIn, Instagram, YouTube, and X.

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: failure to successfully commercialize BRINSUPRI in the U.S. or to maintain U.S. approval for BRINSUPRI; failure to obtain, or delays in obtaining, regulatory approvals for BRINSUPRI in Europe or Japan, including the risk that any regulatory approvals, if granted, may be subject to significant limitations on use or subject to withdrawal or other adverse actions by the applicable regulatory authority; uncertainties in the degree of market acceptance of BRINSUPRI 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 BRINSUPRI 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 obtain adequate reimbursement from government or third-party payors for BRINSUPRI or acceptable prices for BRINSUPRI; development of unexpected safety or efficacy concerns related to BRINSUPRI, including the risk that data generated in further clinical trials of brensocatib may not be consistent with the results of the ASPEN study, which may result in changes to the product label and may adversely affect sales, or result in withdrawal of BRINSUPRI from the market; failure by us to comply with agreements related to brensocatib, including our license agreement with AstraZeneca AB; 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; failure to obtain regulatory approval for potential future brensocatib indications; and 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 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:

Investors

Bryan Dunn

Vice President, Investor Relations

(646) 812-4030

[email protected] 

Media

Claire Mulhearn

Vice President, Corporate Communications

(862) 842-6819

[email protected]   

 

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SOURCE Insmed Incorporated