FDA Grants Orphan Drug Designation to Ikaria® For Use of Inhaled Nitric Oxide in Pulmonary Arterial Hypertension
Investigational New Drug Application Submitted --
HAMPTON, N.J., Jan. 23, 2012 /PRNewswire via COMTEX/ -- 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."
Orphan drug designation is reserved for rare diseases or disorders that affect fewer than 200,000 people in the United States. PAH affects fewer than 200,000 Americans. This designation offers the sponsor tax credits on certain development costs, a waiver of the new drug application (NDA) application user fee, and an exclusivity period of seven years following marketing approval. In addition to the seven-year marketing exclusivity period granted under the orphan drug designation, the INOpulse DS drug-delivery system would be further protected by developing a portfolio of patents, the last of which expires no earlier than 2029.
INOMAX® is a vasodilator, which, in conjunction with ventilator support and other appropriate agents, is indicated for the treatment of term and near-term (>34 weeks gestation) neonates with hypoxic respiratory failure associated with clinical or echocardiographic evidence of pulmonary hypertension, where it improves oxygenation and reduces the need for extracorporeal membrane oxygenation.
INOMAX should not be used in the treatment of neonates known to be dependent on right-to-left shunting of blood. Abrupt discontinuation of INOMAX may lead to a worsening condition. Methemoglobinemia is a dose-dependent side effect of inhaled nitric oxide therapy. Nitrogen dioxide (NO2) forms rapidly in gas mixtures containing nitric oxide and oxygen, and therefore may cause airway inflammation and damage. Methemoglobin, NO2, and FiO2 should be monitored during nitric oxide administration.
Please see attached prescribing information. For additional more information about INOMAX, please visit www.inomax.com .
About Ikaria Inc.
Ikaria, Inc. is a critical care company focused on developing and commercializing innovative therapies designed to address the significant needs of critically ill patients (in the hospital and ICU settings). The company's lead product is INOMAX® (nitric oxide) for inhalation, the only FDA-approved drug for the treatment of hypoxic respiratory failure associated with pulmonary hypertension in term and near-term infants. It is offered through the INOMAX therapy package, an all-inclusive offering of drug product, drug-delivery system, on-site training and 24/7/365 technical assistance and support. The INOMAX therapy package also is marketed in Puerto Rico, Canada, Australia, Mexico and Japan. The company is investigating additional indications for INOMAX in bronchopulmonary dysplasia, and for inhaled nitric oxide with the INOpulse® DS drug-delivery system as a drug-device combination product in pulmonary arterial hypertension (PAH) and chronic obstructive pulmonary disease (COPD). Ikaria's late-stage pipeline is also comprised of LUCASSIN® (terlipressin), a potential treatment for Hepatorenal Syndrome Type 1; as well as Bioabsorbable Cardiac Matrix (BCM), a potential treatment for preventing cardiac remodeling and subsequent congestive heart failure following acute myocardial infarction. Ikaria is headquartered in Hampton, NJ, with a research facility in Madison, WI, and manufacturing facilities in Port Allen, LA and Madison, WI. Please visit www.ikaria.com .