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:6/23/2016)... June 23, 2016 Any dentist who has made ... the current process. Many of them do not even offer ... difficulties and high laboratory costs involved. And those who ARE ... at such a high cost that the majority of today,s ... Dr. Parsa Zadeh , founder of Dental Evolutions Inc. ...
(Date:6/23/2016)... , June 23, 2016 Roche (SIX: ... 510(k) clearance for its Elecsys BRAHMS PCT (procalcitonin) assay ... sepsis or septic shock. With this clearance, Roche is ... a fully integrated solution for sepsis risk assessment and ... with bacterial infection and PCT levels in blood can ...
(Date:6/23/2016)... -- Research and Markets has announced the addition ... to their offering. The ... commercial environment for MedImmune to enter. The US ageing population ... to drive considerable growth for effective anti-influenza medications. The introduction ... considerably, but development is still in its infancy. ...
Breaking Medicine Technology:
(Date:6/26/2016)... ... June 26, 2016 , ... Many women are confused ... endometriosis. These women need a treatment plan to not only alleviate symptoms and ... help for preservation of fertility and ultimately achieving a pregnancy. The specialists at ...
(Date:6/25/2016)... California (PRWEB) , ... June 25, 2016 , ... "With ... fit their specific project," said Christina Austin - CEO of Pixel Film Studios. ... customizable and all within Final Cut Pro X . Simply select a ProHand ...
(Date:6/25/2016)... ... ... On Friday, June 10, Van Mitchell, Secretary of the Maryland Department of Health ... of their exemplary accomplishments in worksite health promotion. , The Wellness at Work Awards ... at the BWI Marriott in Linthicum Heights. iHire was one of 42 businesses to ...
(Date:6/24/2016)... ... ... June 19, 2016 is World Sickle Cell Observance Day. In an effort ... holistic treatments, Serenity Recovery Center of Marne, Michigan, has issued a pain ... Disease (SCD) is a disorder of the red blood cells, which can cause episodes ...
(Date:6/24/2016)... ... June 24, 2016 , ... Dr. Amanda Cheng, ... treatment. Dr. Cheng has extensive experience with all areas of orthodontics, including robotic ... osteogenic orthodontics. , Micro-osteoperforation is a revolutionary adjunct to orthodontic treatment. It ...
Breaking Medicine News(10 mins):