The FDA has granted Orphan Drug designation to AIT-CF, Advanced Inhalation Therapies' (AIT) proprietary high dose formulation of nitric oxide (NO) for adjunctive treatment of cystic fibrosis (CF). In the U.S., Orphan Drug designation provides a variety of incentives, including seven years of market exclusivity, should AIT-CF receive FDA approval.
AIT-CF is a proprietary nitric oxide (NO) formulation and delivery system designed to deliver a high dose formulation (160ppm) to the lungs using positive air pressure and integrated monitoring parameters. The Company's novel 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.
Amir Avniel, CEO of Advanced Inhalation Therapies, said, "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 II, open label, multi-center study of CF patients over 10 years of age demonstrated the safety and efficacy of our product candidate."
Cystic Fibrosis (CF) 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. For hospitalized CF patients, current treatments are supportive through the administration of oxygen through inhalation with bronchiodilators, and include broad spectrum antibiotics and sometimes systemic steroids. Because these treatments do not eliminate all microbes, patients often suffer recurrent infections causing lung damage and ultimately in many cases respiratory failure.
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. A marketing application for a prescription drug product that has received Orphan Drug designation is not subject to the Prescription Drug User Fee Act (PDUFA) fees unless the application includes an additional indication other than the rare disease, or condition for which the therapeutic was not designated.