Navigation Links
Edison Pharmaceuticals Announces FDA Grants EPI-743 Orphan Drug Designation
Date:6/7/2011

MOUNTAIN VIEW, Calif., June 8, 2011 /PRNewswire/ -- Edison Pharmaceuticals, Inc. announced today the United States Food and Drug Administration has granted orphan drug designation to EPI-743 for treatment of inherited mitochondrial respiratory chain diseases. Today, these mitochondrial diseases affect an estimated 60,000 individuals.

The Orphan Drug Act, passed in 1982, provides incentives for companies to pursue treatments for diseases affecting fewer than 200,000 individuals in the United States. Such designation provides an accelerated review and a seven-year period of exclusivity upon FDA approval.

The FDA granted orphan drug designation to Edison's EPI-743 based on review of the application, which included clinical and pre-clinical data demonstrating a favorable efficacy and safety profile.

About Inherited Mitochondrial Disease

Inherited mitochondrial diseases are genetic disorders that share as a common link defects in how cells make and regulate energy. They can arise through defects in genes located in the nucleus or mitochondria. An estimated 2,000 defects in nuclear DNA and 200 defects in mitochondrial DNA have been identified that are pathogenic(1). Inherited mitochondrial diseases are clinically diverse and poorly understood. They can affect virtually any organ system in the body. As the brain and muscle tissue use an extraordinary amount of energy, they are dramatically affected by these disorders. Mitochondrial diseases often present with central nervous system manifestations, but they also result in a variety of other significant clinical signs that include diabetes, heart failure, liver failure, deafness, blindness, renal insufficiency, and muscle weakness and fatigue.

The incidence of mitochondrial disease is estimated at 1-5 in 10,000. However, this number may underestimate the true number of people with mitochondrial disease. While new genetic screening tools have made detection of mitochondrial disease possible, the majority of individuals with a clinical diagnosis of mitochondrial disease may not have a confirmatory genetic diagnosis. Some estimates place the number of clinically diagnosed mitochondrial disease patients without a genetic diagnosis– so called mitochondrial syndromes– at 10 times the incidence of genetically defined disease.

Science today is at the frontier of mitochondrial medicine. Certain diseases that previously lacked a known cause are now being defined as mitochondrial in etiology. It is possible that in the future mitochondrial disease will be implicated in additional diseases, such as in the biology of cancer.

Edison Pharmaceuticals

Edison Pharmaceuticals is a patient- and physician-founded company devoted to developing new medicines that will transform the lives of those diagnosed with rare and neglected diseases. Currently, the company is focused on inherited mitochondrial diseases for which there are no approved drugs. These conditions are life-threatening and highly debilitating. Edison's specialized skill set spans the discovery, development, and translation of redoxbased drugs.

(1) http://www.ninds.nih.gov/news_and_events/proceedings/20090629_mitochondrial.htm


'/>"/>
SOURCE Edison Pharmaceuticals, Inc.
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. Samsung Medison Pledges to Become a World-Class Medical Device Company
2. NOMAD by Aribex Receives Edison Best New Product Award
3. Quest Diagnostics Honored for Diagnostic Innovation at 2011 Edison Awards
4. Osteotechs Plexur M® Technology Wins Thomas Alva Edison Patent Award from the New Jersey Research and Development Council
5. Zargis Medical Strikes Gold at 2010 Edison Awards
6. Orthocare Innovations Compas and Smart Pyramid Recognized at Edison Best New Product Awards
7. Zargis Cardioscan Jointly Named as Finalist in 2010 Edison Best New Product Awards
8. Edison Pharma Hits Milestone as A0001 Enters Phase 1 Clinical Development
9. Topaz Pharmaceuticals Announces New Drug Application for the Treatment of Head Lice Accepted by FDA
10. Aeolus Pharmaceuticals, Inc. Presentation Now Available for On-demand Viewing at RetailInvestorConferences.com
11. Onyx Pharmaceuticals to Present at the Goldman Sachs 32nd Annual Global Healthcare Conference
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/30/2016)... 2016 According to a ... Market by Type (Stability, Raw Materials, Method Validation, ... (Pharmaceutical Companies, Medical Device Companies) - Global Forecast ... healthy growth during the last decade and is ... between 2016 and 2021 to reach USD 4.13 ...
(Date:5/30/2016)... Eye expert s ... seek an eye examination before leaving ...   Moorfields Eye Hospital Dubai, the first overseas branch ... , has identified premature babies as a special concern in the ... vulnerability to retinopathy of prematurity (ROP). ROP is a potentially blinding ...
(Date:5/27/2016)... TEL-AVIV, Israel , May 27, 2016 /PRNewswire/ ... KTOV), an innovative biopharmaceutical company focused on late-stage ... by Dexcel Pharma of pivotal batches ... Food and Drug Administration (FDA). This follows Kitov,s ... pivotal Phase III trial successfully met its primary ...
Breaking Medicine Technology:
(Date:5/31/2016)... ... 2016 , ... TeaZa® Energy, LLC announces the launch of a ... Tobacco Day . The new flavor—Tropical TeaZa? Energy—will be available to customers exclusively ... The new flavor is best described as a juicy, taste bud takeover. A ...
(Date:5/31/2016)... Aviv (PRWEB) , ... May 31, 2016 , ... ... the May 4, 2016 Cool Vendors in Security Infrastructure Protection report by Gartner1 ... and publishes a series of research reports evaluating these innovative vendors and their ...
(Date:5/31/2016)... ... May 31, 2016 , ... Advertising ... firm and statement solutions provider, for the tenth consecutive year as a Top ... of Advertising Age, and SourceLink ranked eighteenth in the “U.S. CRM/Direct Marketing Agency” ...
(Date:5/31/2016)... ... 31, 2016 , ... CURE Media Group , the ... added Cancer and Careers to its Advocacy Spotlight Partnership ... successful careers while fighting cancer. , As partners, both organizations will supply timely ...
(Date:5/30/2016)... Miami, FL (PRWEB) , ... May 30, 2016 , ... ... will means for their children and their efforts to keep their households lice free. ... schools across Hawaii are enacting new policies that keep kids in the classroom despite ...
Breaking Medicine News(10 mins):