Insmed Incorporated announced the European Commission (EC) has granted marketing authorization for ARIKAYCE? Liposomal 590 mg Nebulizer Dispersion for the treatment of nontuberculous mycobacterial (NTM) lung infections caused by Mycobacterium avium complex (MAC) in adults with limited treatment options who do not have cystic fibrosis. Consideration should be given to official guidance on the appropriate use of antibacterial agents.

"We are thrilled that for the first time, non-CF patients with NTM lung infections caused by MAC in the European Union (EU) have an approved therapy to help manage this difficult-to-treat condition, providing a new approach for those who have suffered with few, or no, treatment options," said Will Lewis, Chair and Chief Executive Officer of Insmed. "Today's approval underscores our commitment to serving the MAC lung disease community around the world, and we look forward to bringing ARIKAYCE to appropriate patients in the EU."

The EC approval of ARIKAYCE is based on results from the randomized, open-label, global Phase 3 CONVERT study, which demonstrated that once-daily ARIKAYCE, when combined with multi-drug regimen (MDR), improved sputum culture conversion rates in patients with refractory NTM lung disease caused by MAC compared to MDR therapy alone. The most common side effects with ARIKAYCE affecting the respiratory system are dysphonia, cough, dyspnea, and hemoptysis.

"Today's approval marks a significant milestone in advancing care for patients with MAC lung disease in the EU," said Professor Michael Loebinger, Respiratory Consultant at Royal Brompton Hospital, London, Professor of Practice (Respiratory Medicine) at Imperial College, London, and an investigator in the CONVERT study. "Currently, many patients fail to respond to the standard treatment regimen and continue to suffer from the debilitating effects of this rare and serious disease. Results from the landmark CONVERT study show that adding ARIKAYCE has the potential to help patients who were refractory to standard treatment achieve culture conversion—a critical outcome."

The EC approval follows a positive opinion from the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA) on July 24, 2020. In addition, the Committee for Orphan Medicinal Products of the EMA has adopted a positive opinion recommending that ARIKAYCE maintain Orphan Drug Designation in the EU for the treatment of NTM lung disease, which was originally granted to Insmed in 2014.

Insmed plans to launch ARIKAYCE first in Germany, followed by the United Kingdom (UK) and other EU markets, subject to local reimbursement processes. As part of Insmed's comprehensive approach to patient support, the Company has established country-specific programs to provide patients with direct and ongoing support and information.

"The treatment of MAC lung disease is challenging, with a significant need for new therapies that improve upon the current standard of care and offer options to patients who previously have not been treated successfully," said Marc Lipman, Professor of Respiratory Medicine at University College, London, and one of the founding Trustees of NTM Patient Care UK.

In the United States, ARIKAYCE (under the generic name amikacin liposome inhalation suspension), is the first and only approved treatment for MAC lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. Insmed has submitted a new drug application for ARIKAYCE in Japan for the treatment of patients with NTM lung disease caused by MAC who did not sufficiently respond to prior treatment.

Mycobacterium avium complex (MAC) lung disease is a rare and serious disorder that can significantly increase morbidity and mortality. Patients with MAC lung disease can experience a range of symptoms that often worsen over time, including chronic cough, dyspnea, fatigue, fever, weight loss, and chest pain. In some cases, MAC lung disease can cause severe, even permanent damage to the lungs, and can be fatal. MAC lung disease is an emerging public health concern worldwide with significant unmet need.

ARIKAYCE is approved in the United States as ARIKAYCE? (amikacin liposome inhalation suspension) and in the EU as ARIKAYCE? Liposomal 590 mg Nebuliser Dispersion. Current international treatment guidelines recommend the use of ARIKAYCE for appropriate patients. ARIKAYCE is a novel, inhaled, once-daily formulation of amikacin, an established antibiotic that was historically administered intravenously and associated with severe toxicity to hearing, balance, and kidney function. Insmed's proprietary PULMOVANCE? liposomal technology enables the delivery of amikacin directly to the lungs, where liposomal amikacin is taken up by lung macrophages where the infection resides, while limiting systemic exposure. ARIKAYCE is administered once daily using the Lamira? Nebulizer System manufactured by PARI Pharma GmbH (PARI).