A new treatment for the lung infections that cystic fibrosis patients are prone to has just been granted a special status by US Food and Drug Administration.
The nitric oxide treatment developed by Israel’s Advanced Inhalation Therapies Ltd (AIT), has been granted Orphan Drug designation by the FDA. This 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, which describes CF.
But with its technology, AIT is developing treatments for other lung-related conditions, such as pneumonia and asthma, the company said.
CF is a incurable, hereditary genetic disease that causes the body to develop a mucus (usually in the lungs) that acts as a sponge for bacteria. Germs thrive in the mucus, and lung infections – often life-threatening – are common. Antibiotics, as well as CF-specific drugs, are usually used to treat infections and to keep the body’s airways clear.
That is exactly what its treatment does, AIT said. The treatment uses nitric oxide (not to be confused with nitrous oxide, the “laughing gas” used by dentists), which is a small gas molecule composed of oxygen and nitrogen produced naturally in the body by various cell types, to enhance the body’s immune system. In addition to its physiological role of enhancing blood flow and circulation, said AIT, NO acts as a “killer” molecule as part of the body’s defense mechanism against foreign pathogens such as bacteria, viruses, parasites and fungi.
AIT-CF delivers a high dose (160 ppm) of NO to the lungs using positive air pressure and integrated monitoring parameters. The advantage of its products – and its efficacy compared to other NO-based treatments – is in the delivery, said AIT CEO Amir Avniel.
The system ensures that enough NO gets to all parts of the lungs, eliminating bacteria, viruses, fungi and other microbes. It includes an integrated monitoring system that continuously the patient, to ensure that they are getting a sufficient dose – and tailoring the dose to keep infections away.
“Our system is designed to be user friendly, making the operation and maintenance of the system very intuitive for the medical staff allowing a reduced training time,” he said.
“Receiving Orphan Drug Designation in September 2014 represents an important milestone as we continue to advance the clinical development of AIT-CF,” said Avniel. “Airway phlegm that contains bacterial colonizations and infections is the primary complication of CF. Inhalation of NO has demonstrated anti-infective, anti-inflammatory 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.”