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:4/29/2016)... India , April 29, 2016 ... Pipeline Review, H1 2016" market research report that ... complete with comparative analysis at various stages, therapeutics ... route of administration (RoA) and molecule type, along ... releases. It also reviews key players involved in ...
(Date:4/29/2016)... , April 29, 2016 Glycotope ... expertise, today announces the appointment of Dr. Alfredo ... Zurlo is an oncologist with many years clinical experience ... biotechnology industries. His last role was at Mologen AG ... the Executive Board. Previously Dr. Zurlo held various positions ...
(Date:4/28/2016)...  While Abbott,s announced purchase of St. Jude ... and stent business, healthcare research firm Kalorama Information ... into patient monitoring.  Kalorama said that patient monitoring ... with double-digit growth expected the next 5 years, ... Patient Monitoring . Abbott Laboratories agreed to ...
Breaking Medicine Technology:
(Date:4/29/2016)... ... 30, 2016 , ... Mercy College is expanding its Graduate Business Programs to ... expanding due to high demand: Master of Business Administration (MBA), Master of Organizational Leadership ... , School of Business Graduate Program Chair Dr. Ray Manganelli said: “We believe ...
(Date:4/29/2016)... ... 2016 , ... Memorial Healthcare System Graduate Medical Education ... that it has received accreditation for its residency program on Physical Medicine and ... Memorial is currently pursuing, including Pediatrics and Internal Medicine. This accreditation allows the ...
(Date:4/29/2016)... ... 29, 2016 , ... On Tuesday, April 26, 2016 members of the HomeTown ... of Gov. Nathan Deal on SB 258, the “Rural Health Care Relief” Bill. , ... 70% tax credit to individuals and corporations which donate directly to a “rural hospital” ...
(Date:4/29/2016)... VA (PRWEB) , ... April 29, 2016 , ... ... with student loans more flexibility in repaying their loans, more information about their ... a time when total outstanding student loan debt, including federal and private loans, ...
(Date:4/29/2016)... Fla. (PRWEB) , ... April 29, 2016 , ... ... Hospital surgeon reveals that infants born with severe congenital diaphragmatic hernia have better ... is born with congenital diaphragmatic hernia (CDH)—a condition where the diaphragm fails to ...
Breaking Medicine News(10 mins):