Navigation Links
EMA Grants Orphan Designation to Edison Pharmaceuticals for EPI-743 for Treatment of Leigh Syndrome
Date:9/11/2012

MOUNTAIN VIEW, Calif., Sept. 12, 2012 /PRNewswire/ -- Edison Pharmaceuticals announced today that the Committee for Orphan Medicinal Products (COMP), European Medicines Agency, has granted orphan designation to EPI-743 for the treatment of Leigh syndrome.

In their decision the committee noted, "… the COMP delivered for a first time ever a positive opinion on orphan designation for the treatment for Leigh syndrome, for which no authorized treatments exist in the EU. This syndrome is a very rare and severe disease caused by mutations in mitochondrial respiratory enzymes, leading mainly to neurological deficits and a poor survival for these patients."

Under Expanded Access in the United States, Edison has studied EPI-743 in 80 subjects with mitochondrial disease. Of those subjects treated in the Expanded Access program, 23 had been diagnosed with Leigh syndrome. Favorable data obtained from the treatment of these children, as well as those treated in Europe under both compassionate use and a phase 2A study, formed the basis of the company's COMP orphan designation application.

Results of a recently completed phase 2A study entitled "Prospective Open Label Study of EPI-743 in Children with Leigh Syndrome (Subacute Necrotizing Encephalomyelopathy)" will be announced soon.

About Edison Pharmaceuticals

Edison Pharmaceuticals is a specialty pharmaceutical company dedicated to developing treatments for children with orphan mitochondrial diseases.


'/>"/>
SOURCE Edison Pharmaceuticals
Copyright©2012 PR Newswire.
All rights reserved

Page: 1

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. FDA Grants Orphan Drug Designation To GeNO LLC For Use Of Inhaled Nitric Oxide In Treatment Of Persistant Pulmonary Hypertension Of The Newborn (PPHN)
6. Rep. Fattah Announces More Than $10.7 Million in Grants for Neuroscience, Scientific and Medical Research in Philadelphia
7. U.S. FDA Grants Priority Review to Bedaquiline (TMC207) for Multi-Drug Resistant Tuberculosis Treatment
8. Neuralstem Grants First Licenses For CNS Therapy Surgical Devices
9. Omeros Adds Four More Unlocked Orphan GPCRs to its Portfolio
10. NORD Calls Senate Bill Most Important Since Orphan Drug Act
11. Perrigo Announces FDA Final Approval Of Tris Pharmas Dextromethorphan Polistirex Extended-Release Oral Suspension
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/11/2017)... June 10, 2017  Eli Lilly and Company (NYSE: ... Phase 3 studies of galcanezumab, an investigational treatment for ... data on several key secondary endpoints for galcanezumab compared ... these studies (EVOLVE-1, EVOLVE-2 and REGAIN) will be presented ... meeting in Boston . ...
(Date:6/9/2017)... June 9, 2017 AirXpanders, Inc. (ASX: AXP) ... the design, manufacture, sale and distribution of the AeroForm® ... progress of its commercial roll-out in the ... more than one hundred (100) medical institutions and health ... offers a needle-free alternative for women who choose reconstructive ...
(Date:6/8/2017)...  Less than a month ago, amateur hackers executed ... including hospital networks, in over 150 countries. ... largest online extortion attempts ever recorded. With the increasing ... imperative that providers understand where the risks lie, and ... and many other very real cyber threats.  ...
Breaking Medicine Technology:
(Date:6/24/2017)... ... June 24, 2017 , ... The Pennsylvania Athletic ... Erie Bayfront and Erie Convention Center on June 8-10. The weekend consisted ... quiz bowl, award and scholarship presentations, and professional networking. , On Friday ...
(Date:6/24/2017)... ... June 24, 2017 , ... Doorknobs are for convenience, deadbolts are for ... a guard dog or having an alarm system installed. But unless there is a ... Yair Frenkel, owner of TX Premier Locksmith in Killeen, TX says: “In the majority ...
(Date:6/23/2017)... Long Beach, CA (PRWEB) , ... June 23, 2017 , ... ... (ANRF) has earned its ninth consecutive four-star rating from premier online charity evaluator, Charity ... ANRF in the top 1% of all charities reviewed by Charity Navigator and earns ...
(Date:6/23/2017)... Va. (PRWEB) , ... June 23, 2017 , ... The ... the president's nominee for deputy secretary of Veterans Affairs, retired Marine Col. Thomas G. ... Veterans Affairs. , Bowman brings an intimacy with the issues and challenges ...
(Date:6/23/2017)... ... June 23, 2017 , ... Everybody has their own personal preference when it ... it, and some people don't like it at all. FindaTopDoc took a look at ... Erotic literature can give readers a taste of their deepest, darkest fantasies and has ...
Breaking Medicine News(10 mins):