Navigation Links
FDA Grants Orphan Drug Designation To GeNO LLC For Use Of Inhaled Nitric Oxide In Treatment Of Persistant Pulmonary Hypertension Of The Newborn (PPHN)
Date:8/14/2012

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.Media Contacts:Carla Burigatto, 610-457-2178 Brian Heffron, 781-799-9568 cburigatto@gmail.com bheffron@ctpboston.com
'/>"/>

SOURCE GeNO LLC
Copyright©2012 PR Newswire.
All rights reserved

Related medicine technology :

1. Nektar Announces that FDA Grants Fast Track Designation to NKTR-181, a New Oral Opioid Analgesic Molecule, for the Treatment of Moderate to Severe Chronic Pain
2. Community Foundation Announces $2.6 Million In Grants From Centinela Medical Funds
3. U.S. FDA Grants Priority Review to the New Drug Application for Bayers Regorafenib to Treat Patients with Metastatic Colorectal Cancer
4. European Commission Grants Orphan Medicinal Product Designation for Emmaus Medicals Sickle Cell Treatment
5. Omeros Adds Four More Unlocked Orphan GPCRs to its Portfolio
6. NORD Calls Senate Bill Most Important Since Orphan Drug Act
7. Perrigo Announces FDA Final Approval Of Tris Pharmas Dextromethorphan Polistirex Extended-Release Oral Suspension
8. Tris Pharma Announces Approval of First-Ever Generic to Delsym® Dextromethorphan Polistirex Extended-Release Suspension
9. CSL Behring Receives FDA Orphan Drug Designation for rIX-FP, a Novel Therapy in Development to Treat Congenital Factor IX Deficiency (Hemophilia B)
10. Grifols Initiates Safety Study of Inhaled Alpha1-Proteinase Inhibitor (Human) Following Orphan Drug Designation for Cystic Fibrosis
11. Albireo Receives Positive Opinion for Orphan Drug Designation for A4250 for Severe Liver Diseases
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/2/2016)... On Thursday, the NASDAQ Composite ended the trading ... edged 0.36% higher, to finish at 19,191.93; and the S&P ... as six out of nine sectors ended the day in ... Services equities: Myriad Genetics Inc. (NASDAQ: MYGN ), ... Inc. (NASDAQ: INCR ), and La Quinta Holdings ...
(Date:12/2/2016)... 2016  Today, Simpson Healthcare Executives, a global leader ... selected as winners of multiple awards at the 2016 ... the PLATINUM level, Blue Zones Menu at the GOLD ... Level, and our proprietary 3ConneX Platform at the HONORABLE ... we are excited to showcase our new Platinum and ...
(Date:12/2/2016)... Columbia , December 2, 2016 bioLytical Laboratories ... gegeben, ab diesem Monat seinen  INSTI HIV-Selbsttests  in einer Version mit geringeren ... ... New: INSTI Self Test! (PRNewsFoto/bioLytical ... ,      (Photo: http://photos.prnewswire.com/prnh/20161201/444833 ...
Breaking Medicine Technology:
(Date:12/2/2016)... ... December 02, 2016 , ... More than ... Alabama are expected to attend the UNCF Dothan-Wiregrass Mayor’s Luncheon Dec. 9, 2016. ... for area students and operating support to UNCF-member institutions, including Miles College, Oakwood ...
(Date:12/2/2016)... Raleigh, NC (PRWEB) , ... December 02, 2016 , ... ... the Open Enrollment Period (or Annual Election Period), is ending December 7th. Currently-enrolled Medicare ... plan (Part C) or prescription drug plan (Part D) need to make changes during ...
(Date:12/2/2016)... ... December 02, 2016 , ... Dr. Raffi Hovsepian, a leading ... the 2016 “Guide to America’s Top Plastic Surgeons” for seven consecutive years. The ... education, experience, and professional associations. , One the most frequently honored surgeons ...
(Date:12/2/2016)... ... 02, 2016 , ... The PAINWeekEnd Regional Conference will be ... in Honolulu, offering local frontline clinicians the opportunity to extend their certified continuing ... for supplemental training related to pain management has surged dramatically in recent years, ...
(Date:12/2/2016)... , ... December 02, 2016 , ... Yisrayl Hawkins, at ... into how this current generation fits into Bible Prophecy. Yisrayl says this generation, known ... pointing to this conclusion, showing how the details line up exactly with Bible Prophecy ...
Breaking Medicine News(10 mins):