— 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, NJ, USA I October 28, 2020 I 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.
About ARIKAYCE
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.
U.S. INDICATION
LIMITED POPULATION: ARIKAYCE® is indicated in adults, who have limited or no alternative treatment options, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. As only limited clinical safety and effectiveness data for ARIKAYCE are currently available, reserve ARIKAYCE for use in adults who have limited or no alternative treatment options. This drug is indicated for use in a limited and specific population of patients.
This indication is approved under accelerated approval based on achieving sputum culture conversion (defined as 3 consecutive negative monthly sputum cultures) by Month 6. Clinical benefit has not yet been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
Limitation of Use: ARIKAYCE has only been studied in patients with refractory MAC lung disease defined as patients who did not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. The use of ARIKAYCE is not recommended for patients with non-refractory MAC lung disease.
Patients are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1–800–FDA–1088. You can also call the Company at 1-844-4-INSMED.
Please see Full Prescribing Information.
About Insmed
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.
SOURCE: Insmed
Post Views: 32
— 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, NJ, USA I October 28, 2020 I 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.
About ARIKAYCE
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.
U.S. INDICATION
LIMITED POPULATION: ARIKAYCE® is indicated in adults, who have limited or no alternative treatment options, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. As only limited clinical safety and effectiveness data for ARIKAYCE are currently available, reserve ARIKAYCE for use in adults who have limited or no alternative treatment options. This drug is indicated for use in a limited and specific population of patients.
This indication is approved under accelerated approval based on achieving sputum culture conversion (defined as 3 consecutive negative monthly sputum cultures) by Month 6. Clinical benefit has not yet been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
Limitation of Use: ARIKAYCE has only been studied in patients with refractory MAC lung disease defined as patients who did not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. The use of ARIKAYCE is not recommended for patients with non-refractory MAC lung disease.
Patients are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1–800–FDA–1088. You can also call the Company at 1-844-4-INSMED.
Please see Full Prescribing Information.
About Insmed
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.
SOURCE: Insmed
Post Views: 32
