Ikaria receives FDA orphan drug designation for use of iNO in PAH

Ikaria, Inc., a critical care company focused on developing and commercializing innovative therapies for critically ill patients, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for the use of inhaled nitric oxide (iNO) with the INOpulse® DS drug-delivery system as a combination product for pulmonary arterial hypertension (PAH). An investigational new drug application (IND) for PAH was submitted to the FDA in November 2011, and the company is pursuing clinical development under this open IND.

Nitric oxide is naturally produced by many cells in the human body, and is known as a "signaling molecule" due to its ability to penetrate cell membranes to deliver a signal to nearby muscles to relax.  When inhaled, nitric oxide selectively relaxes the cells of the pulmonary vasculature, resulting in increased blood flow through the lungs and delivery of more oxygenated blood to the body. Inhaled nitric oxide is available as INOMAX® (nitric oxide) for inhalation, a vasodilator, which, in conjunction with ventilation and other appropriate agents, treats term and near-term newborns (>34 weeks gestation) with hypoxic respiratory failure associated with evidence of pulmonary hypertension. 

Based on this use, Ikaria is investigating the use of iNO in patients with PAH, which is hypertension in the arteries between the heart and lungs.  The delivery of iNO will be pulsed to synchronize with the patient's breathing pattern through Ikaria's next-generation INOpulse® DS drug-delivery system, which is specially engineered for use in spontaneously breathing patients.  Ikaria's PAH development program, known as IK-7001, will investigate the use of iNO/INOpulse DS as a drug-device combination product.

"Our receipt of orphan drug designation for the use of iNO via the INOpulse in PAH, combined with the IND we submitted last year, speaks to the solid progress of our late-stage pipeline," stated Daniel Tasse, Chairman and CEO of Ikaria.  "We're delighted to have PAH and bronchopulmonary dysplasia as additional indications under investigation for iNO, and also are planning its investigation with the INOpulse DS in chronic obstructive pulmonary disease."