SOURCE: Insmed Incorporated

March 28, 2013 08:00 ET

ARIKACE Receives Orphan Drug Designation for Treating Infections Caused by Non-Tuberculous Mycobacteria

MONMOUTH JUNCTION, NJ--(Marketwire - Mar 28, 2013) - Insmed Incorporated (NASDAQ: INSM), a biopharmaceutical company focused on developing and commercializing inhaled therapies for patients battling serious lung diseases in orphan indications that are often life-threatening, today announced that ARIKACE®, the Company's Iiposomal amikacin for inhalation, has received orphan drug designation from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development for the treatment of infections caused by non-tuberculous mycobacteria (NTM).

Orphan drug designation provides certain exclusivity benefits, tax credits for certain research and a waiver of the New Drug Application user fee. According to a recent company-sponsored patient chart study conducted by Clarity Pharma Research, approximately 50,000 cases of NTM lung disease were treated by physicians in the U.S. during 2011. There is no current FDA-approved treatment for NTM lung infection.

"The FDA's timely approval of our request for orphan drug designation for ARIKACE to treat non-tuberculous mycobacteria is a key milestone that supports our broader strategy for this potentially life-saving therapy," stated Will Lewis, President and Chief Executive Officer of Insmed. "NTM is a chronic, debilitating disease, and currently available treatments have shown limited efficacy and tolerability. We continue to enroll patients in our phase 2 clinical trial of ARIKACE to treat NTM patients in the U.S. and Canada and look forward to having top-line data by the end of this year."

About Insmed

Insmed Incorporated is a biopharmaceutical company dedicated to improving the lives of patients battling serious lung diseases through the development and commercialization of inhalation therapies in orphan patient populations with critical unmet needs. Insmed's lead candidate, ARIKACE® or liposomal amikacin for inhalation, is engineered to deliver a proven and potent anti-infective directly to the site of serious lung infections to improve the efficacy, safety and convenience of treatment for at least two identified patient populations: cystic fibrosis (CF) patients with Pseudomonas aeruginosa lung infections and patients with non-tuberculous mycobacteria lung infections (NTM). Insmed's phase 3 registrational study of ARIKACE in Europe and Canada completed enrollment and the Company expects top-line clinical results in mid-2013. Insmed's phase 2 clinical trial in patients with NTM is well under way in the U.S. and Canada with clinical results expected in late 2013. For more information, please visit

Forward-Looking Statements

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Contact Information

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    Bruce Voss
    Managing Director