February 10th, 2014
Insmed Incorporated, a biopharmaceutical company focused on developing an inhaled anti-infective to treat patients battling serious lung diseases in orphan indications that are often life-threatening, today announced that the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA) has issued a positive opinion on the Company’s application for orphan designation for ARIKACE, the Company’s liposomal amikacin for inhalation, for the treatment of lung disease caused by nontuberculous mycobacteria (NTM). Orphan drug designation in the European Union (EU) is given to products that are designed for the diagnosis, prevention or treatment of rare diseases that are life-threatening or chronically debilitating.
NTM lung disease is a chronic condition that can lead to progressive inflammation and lung damage, and there is a 40% increase in mortality for patients over 65 years of age.1 Treatment usually involves multi-drug regimens that can be poorly tolerated and have limited effectiveness, especially in patients with severe disease or in those who have failed prior treatment attempts. There is no current treatment approved by the EMA or the U.S. Food and Drug Administration (FDA) for NTM lung disease.
Orphan designation from the EMA provides a number of incentives, including protocol assistance, scientific advice specific for designated orphan medicines, ten years of market exclusivity once the medicine is on the market and potential fee reductions. ARIKACE has also received Orphan Drug, Qualified Infectious Disease Product and Fast Track designations from the FDA for the treatment of NTM lung infections.
Insmed remains on track to report top-line data from a Phase 2 clinical trial of ARIKACE in the U.S. and Canada to treat NTM lung disease by the end of the first quarter 2014