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FDA Breakthrough Therapy Designation: #43 For Insmed

June 18, 2014

Insmed, a New Jersey based biopharmaceutical company, develops targeted therapies for patients with serious orphan lung diseases. In Insmed’s Form 8-K, filed with the US Securities and Exchange Commission (SEC) on June 17th, the company announced that :

“ … the US FDA granted ARIKAYCE, or liposomal amikacin for inhalation, Breakthrough Designation for the treatment of adult patients with Nontuberculous Mycobacterial (NTM) lung disease who are treatment refractory. This designation is based on findings from the Company’s US Phase II clinical trial of ARIKAYCE to treat NTM lung infections. The Company has plans to meet with the FDA to discuss the regulatory pathway for ARIKAYCE.”

Arikayce, Insmed’s lead product candidate, is a once-a-day inhaled antibiotic, that is designed to deliver an anti-infective directly to the location of a serious lung infection. NTM is a chronic illness that affects approximately 50,000 patients in the United States. NTM results in lengthy, repeat hospitalizations.

This is the 43rd Breakthrough Therapy Designation (BTD) announced by a sponsor company and the 1st BTD for Insmed. Arikace (spelling in FDA Orphan Designation Database) has three FDA Orphan Drug Designations (ODDs) :

Num FDA ODD Date Indication
1 03.09.2006 Bronchopulmonary Pseudomonas Aeruginosa Infections In Cystic Fibrosis Patients
2 08.25.2009 Bronchiectasis In Patients With Pseudomonas Aeruginosa/Susceptible Microbial Pathogens
3 03.25.2013 Infections caused by Non-Tuberculous Mycobacteria (NTM)


According to an online article, Insmed’s shares closed at $17.78, up $5.32, or about 43%, after the FDA BTD is announced.



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “Glowing heart (114668824)” by 29cm from Hong Kong (Glowing heart) [CC-BY-SA-2.0] | Wikimedia Commons.

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