Navigation Links
FDA Grants Orphan Drug Designation to Ikaria® For Use of Inhaled Nitric Oxide in Pulmonary Arterial Hypertension

HAMPTON, N.J., Jan. 23, 2012 /PRNewswire/ -- 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  

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

 Company Contact:Media Contact:Samina Bari

Claire SojdaIkaria, Inc.

Tiberend Strategic Advisors, Inc.(908) 238-6372  




SOURCE Ikaria, Inc.
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. FDA Grants 510(k) Clearance to Curos® Disinfecting Port Protector
2. Optimer Pharmaceuticals Reports Inducement Grants Under NASDAQ Listing Rule 5635(c)(4)
3. US Patent Office Grants COPAN Invention of Flocked Swabs
4. The European Commission Grants Marketing Authorization for DIFICLIR™ (fidaxomicin) Tablets for Treatment of Clostridium difficile Infections
5. Watson Confirms Court Grants Preliminary Injunction Preventing Launch of Generic LOVENOX®
6. FDA Awards NIPTE Potential $35 Million in Grants to Reform Drug Manufacturing Process
7. Optimer Pharmaceuticals Reports Inducement Grants Under NASDAQ Listing Rule 5635(c)(4)
8. Pevion Grants CSL Option Right to its Therapeutic Candida Vaccine
9. Muscular Dystrophy Association Awards $13.7 Million for 40 New Research Grants
10. FDA Grants Fast Track Designation to Bayers Investigational Compound Radium-223 Chloride for the Treatment of Castration-Resistant Prostate Cancer That Has Spread to the Bone
11. Echo Therapeutics Announces Multiple Patents and Trademark Grants
Post Your Comments:
(Date:11/26/2015)... November 26, 2015 ... "Self Administration of High Viscosity Drugs" report ... has announced the addition of the "Self ... their offering. --> Research and Markets ... the "Self Administration of High Viscosity Drugs" ...
(Date:11/26/2015)... November 26, 2015 ... the "Radioimmunoassay Market by Type (Reagents ... Industry, Academics, Clinical Diagnostic Labs), Application (Research, ... to 2020" report to their offering. ... the addition of the "Radioimmunoassay Market ...
(Date:11/25/2015)... 25, 2015 ... the "Global Brain Monitoring Devices Market ... --> ) has announced the ... Devices Market 2015-2019" report to their ... ( ) has announced the addition ...
Breaking Medicine Technology:
(Date:11/27/2015)... ... November 27, 2015 , ... CBD College is proud to ... Programs (CAAHEP) awarded accreditation to its Diagnostic Medical Sonography program. CBD College is honored ... one of twelve colleges and universities in the state of California make the cut. ...
(Date:11/26/2015)... ... November 26, 2015 , ... Patients ... central Michigan, have come together on Thanksgiving Day to share the things that ... viewing on the Serenity Point YouTube channel, patients displayed what they wrote on ...
(Date:11/26/2015)... ... ... Jobs in hospital medical laboratories and in the imaging field lead the ... Aureus Medical Group . These fields, as well as travel nursing, ranked ... jobs through the company’s website, , The leading healthcare staffing agency ...
(Date:11/26/2015)... ... ... Indosoft Inc., developer and distributor of the world-class Asterisk based contact center ... reliability. , The new Q-Suite 6 platform is based on the latest Java Enterprise ... a specific piece of software for many key components of the suite. Much of ...
(Date:11/25/2015)... ... November 25, 2015 , ... ... recognized once again for its stellar workplace culture with the company’s Cincinnati office ... , Medical Solutions’ Cincinnati office was named a finalist in Cincinnati Business Courier’s ...
Breaking Medicine News(10 mins):