FDA Grants Orphan Drug Designation To GeNO LLC For Use Of Inhaled Nitric Oxide In Treatment Of Persistant Pulmonary Hypertension Of The Newborn (PPHN)
WALTHAM, Mass., Aug. 14, 2012 /PRNewswire/ -- GeNO LLC (www.genollc.com), a privately held pharmaceutical company developing a line of inhaled nitric oxide drug products, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for its nitric oxide (delivered via the GeNOsyl MVG-2000 delivery system) for the treatment of persistent pulmonary hypertension in newborns (PPHN). The orphan drug designation was granted based on a plausible hypothesis for clinical superiority over the approved nitric oxide product.
Orphan Drug designation is granted by the FDA Office of Orphan Drug Products to novel drugs to treat a rare disease or condition affecting fewer than 200,000 persons in the United States. The designation provides the drug developer with a seven-year period of US marketing exclusivity if the drug is the first of its type approved for the specified indication or if it demonstrates superior safety, efficacy, or a major contribution to patient care versus another drug of its type previously granted the designation for the same indication, as well as with tax credits for clinical research costs, the ability to apply for grant funding, clinical research trial assistance and waiver of filing fees.
"Our goal is to use our proprietary chemistry and delivery apparatus to provide low-dose inhaled NO that provides a level of purity that cannot be attained with currently available treatments," said GeNO President and Founder Dr. David Fine. "We believe our technology has the potential to be a transformative therapy for patients suffering from rare diseases like PPHN for whom there are limited treatment options, and we are committed to advancing the clinical and commercial development of our nitric oxide delivery platforms because of the dramatic benefit they may have on the lives of patients. Orphan Drug Designation of GeNO's drug product for use in treating PPHN in neonates is an important step in this direction."
GeNO is developing three unique nitric oxide delivery platforms: a stand-alone gas cylinder system for hospital and outpatient use, a ventilator-based platform for Intensive Care Unit use, and a pocket-sized ambulatory system for chronic outpatient use. All of the platforms are designed to deliver nitric oxide with an extremely low level of nitrogen dioxide while also addressing the cost, complexity, and lack of portability of approved inhaled nitric oxide delivery systems. GeNO's nitric oxide has the potential for use in the treatment of a multitude of serious pulmonary and cardiac diseases, and potentially could reach a much larger group of patients.
GeNO also recently completed a Phase 2 pilot study of its nitric oxide for use as a diagnostic in Pulmonary Arterial Hypertension (PAH), and is currently performing a dose-escalation trial for the Treatment of Pulmonary Hypertension in patients with PAH and Pulmonary Hypertension secondary to Idiopathic Pulmonary Fibrosis (PH-IPF). Additional study details can be found on http://clinicaltrials.gov/-- ClinicalTrials.gov Identifier NCT01265888. At present, GeNO's other delivery technologies are investigational and are not approved by regulatory authorities.
The first human data on the GeNO technology was presented in April 2012 at the ISHLT Conference, studying the safety of the GeNO system for delivering inhaled nitric oxide to patients being evaluated for cardiac transplant. The GeNO device reproducibly provided nitric oxide with low levels of nitrogen dioxide in air. The nitrogen dioxide levels were considerably below the FDA limit of 3 PPM and OSHA limit of 5 PPM even in the presence of oxygen which is known to rapidly convert nitric oxide to nitrogen dioxide.
ABOUT GENO LLC
GeNO LLC, founded in 2006, is a privately held pharmaceutical company that is developing innovative nitric oxide (NO) generation and delivery platforms to enable the true potential of inhaled nitric oxide to be realized for the treatment of a multitude of diseases. The company is focused on applying its technology to delivering inhaled NO to patients in a variety of settings, including ambulatory and in-home use for potential chronic treatment. GeNO will provide stand-alone medical devices and combination products that include the base delivery system and GeNO's nitric oxide gas. GeNO's systems have been designated as Combination Products by the FDA Office of Combination Products, with the Division of Cardiovascular and Renal Products designated as the lead reviewer for GeNO's initial INDs. The combination products will be regulated by the FDA as new drugs with their own NDAs.
SOURCE GeNO LLC