Navigation Links
Edison Pharmaceuticals to Provide Expanded Access to EPI-743 for Mitochondrial Disease
Date:6/8/2011

MOUNTAIN VIEW, Calif., June 8, 2011 /PRNewswire/ -- Edison Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has allowed an Expanded Access program to provide EPI-743 to seriously ill patients diagnosed with inherited respiratory chain diseases of the mitochondria. Patients with genetically confirmed disease and patients who meet specific clinical criteria, absent genetic confirmation are both eligible.

As of June 1, 2011, 40 seriously ill subjects diagnosed with genetically confirmed inherited mitochondrial disease have been treated with EPI-743 worldwide for a cumulative exposure of over 7,436 days. No significant drug-related adverse events have been observed. Initial clinical, biomarker, and brain-imaging data are encouraging. The company cautions that the data obtained in these initial studies is preliminary and needs verification in controlled prospective trials. Edison and its clinical investigator team are working closely with FDA and the European Medicines Agency to expedite prospective phase 2B/3 clinical trials. EPI-743 has received orphan designation from FDA.

Clinical Trial Sites

United States

Clinical trial enrollment sites have been established in North America at Lucile Packard Children's Hospital– Stanford University Medical Center; Akron Children's Hospital; Columbia University; Seattle Children's Hospital; Medical University of South Carolina; Children's Hospital of Philadelphia; and University of California, Los Angeles.

Additional trial sites are being established in Europe, Japan, and in North America.

Treatment Information

Edison Pharmaceuticals

— EdisonPharma.com

United Mitochondrial Disease Foundation

— UMDF.org

Friedreich's Ataxia Research Alliance

— CureFA.org

MitoAction

— MitoAction.org

United States Food and Drug Administration

— Clinicaltrials.gov

EPI-743

EPI-743 is an orally absorbed small molecule that readily crosses into the central nervous system. It works by targeting an enzyme NADPH quinone oxidoreductase 1 (NQO1). Its mode of action is to synchronize energy generation in mitochondria with the need to counter cellular redox stress(1). EPI-743 is in phase 2B/3 pivotal clinical trials.

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(2).

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 to 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) Shrader, W.D.; Amagata, A.; Barnes, A.; Enns, G.M.; Hinman, A.; Jankowski, O.; Kheifets, V.; Komatsuzaki, R.; Lee, E.; Mollard, P.; Murase, K.; Sadun, A.A.; Thoolen, M.; Wesson, K.; Miller, G.; alpha-Tocotrienol quinone modulates oxidative stress response and the biochemistry of aging. Bioorganic Medicinal Chemistry Letters, 2011;21(12):3693-3698.

(2) 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. Edison Pharmaceuticals Announces FDA Grants EPI-743 Orphan Drug Designation
2. Samsung Medison Pledges to Become a World-Class Medical Device Company
3. NOMAD by Aribex Receives Edison Best New Product Award
4. Quest Diagnostics Honored for Diagnostic Innovation at 2011 Edison Awards
5. Osteotechs Plexur M® Technology Wins Thomas Alva Edison Patent Award from the New Jersey Research and Development Council
6. Zargis Medical Strikes Gold at 2010 Edison Awards
7. Orthocare Innovations Compas and Smart Pyramid Recognized at Edison Best New Product Awards
8. Zargis Cardioscan Jointly Named as Finalist in 2010 Edison Best New Product Awards
9. Edison Pharma Hits Milestone as A0001 Enters Phase 1 Clinical Development
10. Topaz Pharmaceuticals Announces New Drug Application for the Treatment of Head Lice Accepted by FDA
11. Aeolus Pharmaceuticals, Inc. Presentation Now Available for On-demand Viewing at RetailInvestorConferences.com
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:4/19/2017)... Medical Corporation (TSX: CRH) (NYSE MKT: CRHM) (the "Company"), announces that it ... 2017 at the Sheraton Hotel in Toronto, Ontario ... the Company is scheduled to present on Tuesday, May 2 at ... Chairman of the Board, Tony Holler will also attend ... For more details ...
(Date:4/19/2017)... , April 19, 2017 Global ... This report on the prostate cancer therapeutics market ... global market. Increasing prevalence of prostate cancer, launch ... in the development of new drugs & therapeutic ... cancer drug due to lesser side effects are ...
(Date:4/19/2017)... , April 19, 2017  New research provides evidence ... advanced Parkinson,s, according to a study released today that will ... Annual Meeting in Boston , April ... to the treatment of Parkinson,s disease, the oral drug levodopa ... life and longevity. But as the disease progresses, the effects ...
Breaking Medicine Technology:
(Date:4/28/2017)... york (PRWEB) , ... April 28, 2017 , ... ... to announce that Aditya Patel M.D. has joined the revolutionary endoscopic practice under ... and board certification in Interventional Pain Medicine. The patented, revolutionary eDiscSculpt Technique created ...
(Date:4/28/2017)... ... April 28, 2017 , ... Early detection and ... drug safety and minimize the cost of development. In this webinar, sponsored by ... cell lines and for cardiac toxicity using induced pluripotent stem cells (iPSCs). , ...
(Date:4/28/2017)... ... April 28, 2017 , ... Researchers at ... ActiGraph’s CentrePoint Data Hub in a sample of participants enrolled in ... activity and sleep monitoring solutions for the global scientific community. The company’s new ...
(Date:4/28/2017)... ... April 28, 2017 , ... NuevaCare, a leading home ... as Millbrae, Burlingame, and Palo Alto, is proud to announce information upgrades to its ... bookmark and read organized content on topics such as home care (generally) as well ...
(Date:4/27/2017)... ... April 27, 2017 , ... Today, ... based in Denver, Colorado, announced an agreement to create the Jane and Leonard ... of the Jane and Leonard Korman Family Foundation. The collaboration leverages the strengths ...
Breaking Medicine News(10 mins):