Early on October 28, 2020, Insmed Inc. (“Insmed”) announced that it received Marketing Authorization from the European Commission for ARIKAYCE. This therapy is designed for adult patients with nontuberculous mycobacterial (NTM) lung infections or lung diseases. However, it is not designed for patients with NTM lung infections who also have cystic fibrosis. Generally, there are few treatments for patients with NTM lung disease within Europe, so ARIKAYCE, administered via nebulizer, fills an unmet need.
Also known as amikacin liposome inhalation suspension or ARIKAYCE Liposomal 590 mg Nebuliser Dispersion, ARIKAYCE is administered through the proprietary Lamira Nebulizer System. PARI Pharma GmbH developed the Lamira system. While the treatment is already approved for use in the United States, the recent European approval was based on data from the Phase 3 CONVERT trial. During the trial, patients received ARIKAYCE 1x daily alongside a multi-drug regimen. Researchers found that using ARIKAYCE in conjunction with other therapies significantly improved outcomes in patients with NTM lung disease. Because of the proprietary PULMOVANCE technology used, ARIKAYCE is also administered directly to the lungs.
While ARIKAYCE is generally well-tolerated, side effects were observed. These include cough, shortness of breath, a hoarse voice, and coughing up blood.
NTM Lung Disease
Nontuberculous mycobacterial lung disease, or NTM lung disease, is a chronic lung infection caused by mycobacteria. According to the CDC:
Nontuberculous mycobacteria (NTM) are mycobacteria other than M. tuberculosis (the cause of tuberculosis) and M. leprae (the cause of leprosy). NTM are environmental organisms that can be found in soil, dust, and water including natural water sources (such as lakes, rivers, and streams) and municipal water sources (such as water that people drink or shower in).
Admittedly, many people are exposed to NTM, sometimes even daily. But a majority of these people do not get sick. However, NTM are more likely to negatively impact those with underlying health conditions, other respiratory ailments, or those who are immunocompromised. NTM lung disease often affects older patients. Symptoms, if they appear, include:
- Coughing up blood
- An intense and persistent cough
- Shortness of breath and/or difficulty breathing
- Night sweats
- Unintended weight loss
- Excess mucus production
- Recurring respiratory, lymph node, skin, or bone infections