FINAL Insmed Receives Positive CHMP Opinion for ARIKAYCE Liposomal 590 mg Nebuliser Dispersion for the Treatment of NTM Lung Infections Caused by MAC in Non-CF Patients with Limited Treatment Options -If Approved, ARIKAY

Insmed Receives Positive CHMP Opinion

for ARIKAYCE Liposomal 590 mg Nebuliser

Dispersion for the Treatment of NTM Lung Infections Caused by MAC in Non-CF Patients with

Limited Treatment Options

-If Approved, ARIKAYCE Will Be

First and Only Therapy in the European Union for This Difficult-to-Treat Condition-

-Decision from European Commission

Expected Second Half of 2020-

BRIDGEWATER, N.J., July 24, 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 Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency

has adopted a positive opinion recommending ARIKAYCE Liposomal 590 mg Nebuliser Dispersion for the treatment of non-tuberculous

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

The European Commission (EC) will review the CHMP opinion, with

a final decision anticipated in the second half of 2020.

"Today's positive opinion from the CHMP marks an

important step in our journey to transform the way MAC lung disease is managed for patients around the world. If approved by the

EC, ARIKAYCE would be the first therapy in both the European Union and the United States for patients with this chronic, debilitating

condition," said Will Lewis, Chairman and Chief Executive Officer of Insmed. "We look forward to potentially bringing

ARIKAYCE to appropriate patients in Europe as quickly as possible."

The CHMP opinion is based on results from the 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 positive CHMP opinion underscores the potential benefit

of ARIKAYCE in the management of MAC lung disease for patients who are particularly difficult to treat and continue to suffer in

the absence of an approved therapy in Europe," said Prof. Dr. Christoph Lange, Medical Director of Clinical Infectious Diseases

at the Medical Clinic of the Research Center Borstel, Leibniz Lung Center, Germany. "As demonstrated in the Phase 3 CONVERT

study, ARIKAYCE has the potential to improve culture-conversion rates in patients with MAC lung disease who were not responsive

to standard of care-a critical milestone in the treatment landscape of this disease."

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 also 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. New international treatment guidelines issued in July 2020 recommend the use of ARIKAYCE in combination

with a multidrug regimen in patients with MAC lung disease who have failed standard therapy after at least six months of treatment.

ARIKAYCE previously received Orphan Drug Designation in the

European Union for the treatment of NTM lung infections.

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.

In the United States, ARIKAYCE is the

first and only FDA-approved therapy indicated for the treatment of Mycobacterium avium complex (MAC) lung disease

as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. Current

international treatment guidelines recommend the use of ARIKAYCE in combination with a multidrug regimen in patients with MAC lung

disease who have failed standard therapy after at least six months of treatment. 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

ARIKAYCE is not approved in any country other than the United

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 liquid medications, including liposomal formulations such as 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.

About CONVERT (INS-212)

CONVERT was a randomized, open-label,

global Phase 3 study designed to confirm the sputum culture conversion results seen in Insmed's Phase 2 clinical study of ARIKAYCE

in patients with refractory NTM lung disease caused by MAC. CONVERT was conducted in 18 countries at more than 125 sites. In the

U.S., the primary efficacy endpoint was the proportion of patients who achieved sputum culture conversion at Month 6 in the ARIKAYCE

plus MDR arm compared to the MDR-only arm. Patients who achieved sputum culture conversion by Month 6 continued in the CONVERT

study for an additional 12 months of treatment following the first monthly negative sputum culture. The CONVERT study also evaluated

the proportion of patients who maintained sputum culture conversion 3 months off all MAC treatment, which was the primary efficacy

IMPORTANT SAFETY INFORMATION FOR

ARIKAYCE IN THE U.S.

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.

Hemoptysis has 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

Bronchospasm has 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

Ototoxicity has 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