European Commission Grants Marketing Authorization for ARIKAYCE® Liposomal 590 mg Nebuliser Dispersion for the Treatment of NTM Lung Infections Caused by MAC in Adult Non-CF Patients with Limited Treatment Options
— ARIKAYCE is the First and Only Therapy Approved in Both the European Union and United States for This Difficult-To-Treat Condition —
— Planned Product Launches to Begin in Germany, Followed by UK and Other EU Countries —
BRIDGEWATER, N.J., Oct. 28, 2020 /PRNewswire/ -- Insmed Incorporated (Nasdaq: INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, today announced that the European Commission (EC) has granted marketing authorization for ARIKAYCE® Liposomal 590 mg Nebuliser Dispersion ("ARIKAYCE") 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," noted 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.
About MAC Lung Disease
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).
About PARI Pharma and the Lamira® Nebulizer System
ARIKAYCE is delivered by a novel inhalation device, the Lamira® Nebulizer System, developed by PARI. Lamira® is a quiet, portable nebulizer that enables efficient aerosolization of ARIKAYCE via a vibrating, perforated membrane. Based on PARI's 100-year history working with aerosols, PARI is dedicated to advancing inhalation therapies by developing innovative delivery platforms and new pharmaceutical formulations that work together to improve patient care.
Insmed Incorporated is a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases. Insmed's first commercial product, ARIKAYCE® (amikacin liposome inhalation suspension), is the first and only therapy approved in the United States for the treatment of refractory Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. MAC lung disease is a chronic, debilitating condition that can cause severe and permanent lung damage. Insmed's earlier-stage clinical pipeline includes brensocatib, a novel oral reversible inhibitor of dipeptidyl peptidase 1 with therapeutic potential in non-cystic fibrosis bronchiectasis and other inflammatory diseases, and treprostinil palmitil, an inhaled formulation of a treprostinil prodrug that may offer a differentiated product profile for rare pulmonary disorders, including pulmonary arterial hypertension. For more information, visit www.insmed.com.
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 timing discussed, projected, anticipated or indicated in any forward-looking statements. Such risks, uncertainties and other factors include, among others, the following: failure to obtain, or delays in obtaining, regulatory approvals for ARIKAYCE outside the U.S. and the European Union or for the Company's product candidates in the U.S., Europe, Japan or other markets, including the United Kingdom as a result of its recent exit from the European Union; failure to successfully commercialize or maintain U.S. approval for ARIKAYCE, the Company's only approved product; business or economic disruptions due to catastrophes or other events, including natural disasters or public health crises; impact of the novel coronavirus (COVID-19) pandemic and efforts to reduce its spread on the Company's business, employees, including key personnel, patients, partners and suppliers; uncertainties in the degree of market acceptance of ARIKAYCE by physicians, patients, third-party payors and others in the healthcare community; the Company's inability to obtain full approval of ARIKAYCE from the FDA, including the risk that the Company will not timely and successfully complete the study to validate a PRO tool and complete the confirmatory post-marketing study required for full approval of ARIKAYCE; inability of the Company, PARI or the Company's other third party manufacturers to comply with regulatory requirements related to ARIKAYCE or the Lamira® Nebulizer System; the Company's inability to obtain adequate reimbursement from government or third-party payors for ARIKAYCE or acceptable prices for ARIKAYCE; development of unexpected safety or efficacy concerns related to ARIKAYCE; inaccuracies in the Company's estimates of the size of the potential markets for ARIKAYCE 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 create an effective direct sales and marketing infrastructure or to partner with third parties that offer such an infrastructure for distribution of ARIKAYCE; failure to obtain regulatory approval to expand ARIKAYCE's indication to a broader patient population; failure to successfully conduct future clinical trials for ARIKAYCE; failure of third parties on which the Company is dependent to manufacture sufficient quantities of ARIKAYCE or the Company's product candidates for commercial or clinical needs, to conduct the Company's clinical trials, or to comply with laws and regulations that impact the Company's business or agreements with the Company; the Company's inability to attract and retain key personnel or to effectively manage the Company's growth; the Company's inability to adapt to its highly competitive and changing environment; the Company's limited experience operating internationally; and changes in laws and regulations applicable to the Company's business, including any pricing reform, and failure to comply with such laws and regulations.
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, 2019, the Company's Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 and any subsequent Company filings with the Securities and Exchange Commission (SEC).
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Laura Perry or Heather Savelle
Senior Director, Corporate Communications