Israel’s Advanced Inhalation Therapies Ltd (AIT), a company which develops therapies that utilize the gas Nitric Oxide, announced that the U.S. Food and Drug Administration granted Orphan Drug Designation to AIT-CF, the Company’s proprietary high dose formulation of nitric oxide (NO) for adjunctive treatment of cystic fibrosis.
Don’t confuse nitric oxide with nitrous oxide(N2O), or laughing gas, which dentists use as a pain killer and many people use as a recreational drug. The first has only one oxygen molecule and the second has two.
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In the U.S., Orphan Drug Designation provides a variety of incentives, including seven years of market exclusivity, should AIT-CF receive FDA approval.
Cystic Fibrosis is a lifelong, hereditary disease that causes mucus to form in the lungs and other organs. In 90% of CF cases, this mucus blocks the airways in the lungs, making it hard to breathe and leading to serious lung infections.
The company describes AIT-CF as a proprietary nitric oxide formulation and delivery system designed to deliver a high dose formulation (160 ppm) to the lungs using positive air pressure and integrated monitoring parameters.
AIT says that its system has the potential to eliminate microbial infections including bacteria, fungi and viruses. NO is produced naturally by the body as a highly effective antimicrobial defense mechanism, but to date no delivery system has been able to deliver an effective and non-toxic antimicrobial dosage to the lungs. AIT’s unique and proprietary system continuously monitors safety and efficacy parameters in the patient and is adaptable to treat a wide range of lung infections.
Mr. Amir Avniel, Chief Executive Officer of Advanced Inhalation Therapies, commented, “Receiving Orphan Drug Designation in September 2014 represents an important milestone as we continue to advance the clinical development of AIT-CF. Airway phlegm that contains bacterial colonizations and infections is the primary complication of CF. Inhalation of NO has demonstrated anti-infective, anti-inflammatory and vasodilating properties in CF patients. The results from our completed Phase 2 open label, multi-center study of CF patients over 10 years of age demonstrated the safety and efficacy of our product candidate.”
The Orphan Drug designation is granted to development-stage novel therapeutics that offer potential value in the treatment of rare diseases and medical conditions that affect fewer than 200, 000 patients in the US. Orphan Drug designation qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act, including tax credits for qualified clinical testing.