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 - ARIKA

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

- Planned Product Launches to

Begin in Germany, Followed by UK and Other EU Countries -

BRIDGEWATER, N.J., October 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

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

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.

IMPORTANT SAFETY INFORMATION FOR

ARIKAYCE IN THE U.S.

WARNING: RISK OF INCREASED RESPIRATORY

ARIKAYCE has been associated with

an increased risk of respiratory adverse reactions, including hypersensitivity pneumonitis, hemoptysis, bronchospasm, and exacerbation

of underlying pulmonary disease that have led to hospitalizations in some cases.

Hypersensitivity Pneumonitis has

been reported with the use of ARIKAYCE in the clinical trials. Hypersensitivity pneumonitis (reported as allergic alveolitis,

pneumonitis, interstitial lung disease, allergic reaction to ARIKAYCE) was reported at a higher frequency in patients treated

with ARIKAYCE plus background regimen (3.1%) compared to patients treated with a background regimen alone (0%). Most patients

with hypersensitivity pneumonitis discontinued treatment with ARIKAYCE and received treatment with corticosteroids. If hypersensitivity

pneumonitis occurs, discontinue ARIKAYCE and manage patients as medically appropriate.

been reported with the use of ARIKAYCE in the clinical trials. Hemoptysis was reported at a higher frequency in patients treated

with ARIKAYCE plus background regimen (17.9%) compared to patients treated with a background regimen alone (12.5%). If hemoptysis

occurs, manage patients as medically appropriate.

been reported with the use of ARIKAYCE in the clinical trials. Bronchospasm (reported as asthma, bronchial hyperreactivity, bronchospasm,

dyspnea, dyspnea exertional, prolonged expiration, throat tightness, wheezing) was reported at a higher frequency in patients

treated with ARIKAYCE plus background regimen (28.7%) compared to patients treated with a background regimen alone (10.7%).

If bronchospasm occurs during the use of ARIKAYCE, treat patients as medically appropriate.

Exacerbations of underlying

pulmonary disease has been reported with the use of ARIKAYCE in the clinical trials. Exacerbations of underlying

pulmonary disease (reported as chronic obstructive pulmonary disease (COPD), infective exacerbation of COPD, infective exacerbation

of bronchiectasis) have been reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (14.8%)

compared to patients treated with background regimen alone (9.8%). If exacerbations of underlying pulmonary disease

occur during the use of ARIKAYCE, treat patients as medically appropriate.

Anaphylaxis and Hypersensitivity

Reactions: Serious and potentially life-threatening hypersensitivity reactions, including anaphylaxis,

have been reported in patients taking ARIKAYCE. Signs and symptoms include acute onset of skin and mucosal tissue hypersensitivity

reactions (hives, itching, flushing, swollen lips/tongue/uvula), respiratory difficulty (shortness of breath, wheezing, stridor,

cough), gastrointestinal symptoms (nausea, vomiting, diarrhea, crampy abdominal pain), and cardiovascular signs and symptoms of

anaphylaxis (tachycardia, low blood pressure, syncope, incontinence, dizziness). Before therapy with ARIKAYCE is instituted, evaluate

for previous hypersensitivity reactions to aminoglycosides. If anaphylaxis or a hypersensitivity reaction occurs, discontinue

ARIKAYCE and institute appropriate supportive measures.

been reported with the use of ARIKAYCE in the clinical trials. Ototoxicity (including deafness, dizziness, presyncope, tinnitus,

and vertigo) were reported with a higher frequency in patients treated with ARIKAYCE plus background regimen (17%) compared to

patients treated with background regimen alone (9.8%). This was primarily driven by tinnitus (7.6% in ARIKAYCE plus background

regimen vs 0.9% in the background regimen alone arm) and dizziness (6.3% in ARIKAYCE plus background regimen vs 2.7% in the background