Insmed Receives Orphan Medical Product Designation for Rare Lung Infection Treatment

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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.

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.

“The EMA’s orphan drug designation represents another step forward in our progress towards bringing this important therapy to the approximately 30,000 patients in the European Union in need of an effective therapy to treat NTM lung disease. We have already commenced the Scientific Advice Working Party process with the EMA to discuss a regulatory path forward for ARIKACE to treat NTM lung disease in Europe… We are actively developing a strategy to commercialize ARIKACE globally in this uncontested orphan disease market, and the EMA’s decision is an important milestone in support of these efforts.”Will Lewis, President and Chief Executive Officer of Insmed

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

See the full press release here.
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