Pipeline and Diseases
Insmed is advancing a pipeline to bring potential new treatments to patients with rare and serious diseases who currently have few, if any, treatment options. We are working with a profound sense of urgency to deliver creative applications of science and technology that have the potential to address these unmet needs.
Product Pipeline
Our research and development pipeline has the potential to address the significant unmet needs of patients with serious and rare diseases.
Nontuberculous Mycobacterial (NTM) Lung Disease
| Preclinical | Phase 1 | Phase 2 | Phase 3 | Approved |
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ARIKAYCE® (Amikacin Liposome Inhalation Suspension)
Refractory NTM: M. avium complex (MAC) -
Nontuberculous mycobacterial (NTM) lung disease is a rare and serious disorder most commonly caused by a group of bacteria called Mycobacterium avium complex (MAC).1 People who have bronchiectasis, COPD, and asthma are at greater risk of getting NTM lung disease.2,3,4
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ARIKAYCE® (Amikacin Liposome Inhalation Suspension) Lifecycle Management
Front Line Label Expansion -
Nontuberculous mycobacterial (NTM) lung disease is a rare and serious disorder most commonly caused by a group of bacteria called Mycobacterium avium complex (MAC).1 People who have bronchiectasis, COPD, and asthma are at greater risk of getting NTM lung disease.2,3,4
Neutrophil-Driven Inflammatory Conditions
| Preclinical | Phase 1 | Phase 2 | Phase 3 | Approved |
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Brensocatib: DPP1 Inhibitor
Non-Cystic Fibrosis Bronchiectasis -
Non-cystic fibrosis bronchiectasis (NCFBE) is a chronic pulmonary disorder in which the bronchi become permanently dilated due to a vicious cycle of inflammation and complications from prior infections.5,6 The condition is marked by frequent pulmonary exacerbations requiring antibiotic therapy and/or hospitalizations.
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Brensocatib: DPP1 Inhibitor
Cystic Fibrosis -
Cystic fibrosis (CF) is a rare, progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time.7 CF is caused by mutations in the cystic fibrosis transmembrane regulator (CFTR) gene.8
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Brensocatib: DPP1 Inhibitor
COVID-19 (IIR) -
COVID-19 is an infectious disease caused by a newly discovered coronavirus. While most people infected with the virus will experience mild to moderate respiratory illness, those with certain risk factors are more likely to develop serious illness.9
Additional Rare Pulmonary Diseases
| Preclinical | Phase 1 | Phase 2 | Phase 3 | Approved |
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Treprostinil Palmitil Inhalation Powder
Pulmonary Arterial Hypertension (PAH) -
Pulmonary arterial hypertension (PAH) is a serious, progressive, rare disease involving narrowing and constriction of the pulmonary arteries that carry blood from the right side of the heart to the lungs.10 PAH is also known as WHO Group 1 Pulmonary Hypertension.
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Treprostinil Palmitil Inhalation Powder
Pulmonary Hypertension associated with Interstitial Lung Diseases (PH-ILD) -
Interstitial lung diseases (ILD) comprise a large group of diseases that cause fibrosis (scarring) of the lungs. Pulmonary hypertension (high blood pressure in the lungs) is a common and important complication of several ILDs that is associated with reduced exercise capacity and poor prognosis.11,12 PH-ILD is also known as WHO Group 3 Pulmonary Hypertension.
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Treprostinil Palmitil Inhalation Powder
Idiopathic Pulmonary Fibrosis (IPF) -
Idiopathic pulmonary fibrosis (IPF) is a serious, chronic, progressive, rare disease in which tissue surrounding the air sacs in the lungs becomes thick and stiff, leading to permanent fibrosis (scarring), which, over time, makes breathing more difficult.13
Internal R&D
| Preclinical | Phase 1 | Phase 2 | Phase 3 | Approved |
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Internal R&D
Various indications -
Insmed conducts research in-house to potentially bring forth new medicines and technologies that address rare and serious diseases.
* In the U.S., as a condition of accelerated approval, Insmed is conducting an additional clinical study to support full approval. Full approved has been granted by the European Commission.
References
- 1 Adjemian J, Prevots DR, Gallagher J, Heap K, Gupta R, Griffith D. Lack of adherence to evidence-based treatment guidelines for nontuberculous mycobacterial lung disease. Ann Am Thorac Soc. 2014;11(1):9-16.
- 2 Mirsaeidi M, Hadid W, Ericsoussi B, Rodgers D, Sadikot RT. Non-tuberculous mycobacterial disease is common in patients with non-cystic fibrosis bronchiectasis. Int J Infect Dis.2013;17(11):e1000-e1004.
- 3 Andréjak C, Nielsen R, Thomsen VØ, Duhaut P, Sørensen HT, Thomsen RW. Chronic respiratory disease, inhaled corticosteroids and risk of non-tuberculous mycobacteriosis. Thorax. 2013;68(3):256-262.
- 4 Fritscher LG, Marras TK, Bradi AC, Fritscher CC, Balter MS, Chapman KR. Nontuberculous mycobacterial infection as a cause of difficult-to-control asthma: a case-control study. Chest. 2011;139(1):23-27.
- 5 Prevalence and incidence of noncystic fibrosis bronchiectasis among US adults in 2013 (Weycker). https://www.ncbi.nlm.nih.gov/pubmed/28555504. Accessed December 2020.
- 6 Advances in bronchiectasis: endotyping, genetics, microbiome, and disease heterogeneity (Flume). https://www.ncbi.nlm.nih.gov/pubmed/30215383. Accessed December 2020.
- 7 Cystic Fibrosis Foundation. About Cystic Fibrosis. https://www.cff.org/What-is-CF/About-Cystic-Fibrosis/. Accessed December 2020.
- 8 National Institutes of Health. Genetic and Rare Disease Information Center. https://rarediseases.info.nih.gov/diseases/6233/cystic-fibrosis. Accessed October 2020
- 9 World Health Organization. Coronavirus. https://www.who.int/health-topics/coronavirus#tab=tab_1. Accessed December 2020.
- 10 Pulmonary Hypertension Association. About Pulmonary Hypertension (PH) and Pulmonary Arterial Hypertension (PAH). http://www.ourphlibrary.com/module1-about-pah/what-is-pah. Accessed January 2021.
- 11 American Lung Association. Interstitial Lung Disease. https://www.lung.org/lung-health-diseases/lung-disease-lookup/interstitial-lung-disease. Accessed January 2021.
- 12 Caminati A. Pulmonary hypertension in chronic interstitial lung diseases. European Respiratory Review 2013 22: 292-301 https://err.ersjournals.com/content/22/129/292. Accessed January 2021.
- 13 National Heart, Lung, and Blood Institute. Idiopathic Pulmonary Fibrosis. https://www.nhlbi.nih.gov/health-topics/idiopathic-pulmonary-fibrosis. Accessed January 2021.
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