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:5/31/2016)... Deutschland und GERMANTOWN, Maryland ... Referenzdatensatz von Horizon bestätigt nachgewiesene ... s   QIAGEN N.V. ... Standard: QIA) gab heute die Ergänzung seines ... und Verifizierungsdaten bekannt, die die bewährte Genauigkeit ...
(Date:5/31/2016)... HILDEN , Germany and ... QIAGEN N.V. (NASDAQ: ... an extensive package consisting of a quality control set ... NGS System, reinforcing the proven accuracy of its next-generation ... and training for laboratories. QIAGEN has incorporated the Horizon ...
(Date:5/31/2016)... , May 31, 2016 Diplomat Pharmacy, Inc. ... Dialogue," a video conversation slated for June 7, 2016, to ... hosted live from noon to 1 p.m. ET, will focus ... helped him along the road to wellness. ... conversations planned between patients and health care professionals, talking about ...
Breaking Medicine Technology:
(Date:5/31/2016)... York, New York (PRWEB) , ... May 31, 2016 , ... ... effects research to provide the best accessories to avoid excessive Smartphone radiation implicated in ... of the study released on the bioRxiv preprint server on May 26th ...
(Date:5/31/2016)... ... June 01, 2016 , ... Psoriasis: Targets and Therapy ... , As corresponding author Professor Luigi Naldi says “The review in Psoriasis: Targets ... Smoking influences the onset and the severity of psoriasis, and negatively influences the ...
(Date:5/31/2016)... ... May 31, 2016 , ... Fire ... CDC, has joined the Fire Door Solutions’ team. Keith will serve Fire ... and frame in-field re-labeling. He will be instrumental in developing educational curriculum ...
(Date:5/31/2016)... ... May 31, 2016 , ... ... that considers individuals’ genetic characteristics and the physical and behavioral worlds in ... in sync. In personalized medicine, diagnosing an individual’s disease depends on accurately ...
(Date:5/31/2016)... CA (PRWEB) , ... May 31, 2016 , ... Throughout the day, adults often find ... sustained motivation and awareness, but what they could be reaching for instead is a cup ... plants that are grown in Japan. This sacred tea is harvested from early June to ...
Breaking Medicine News(10 mins):