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:11/24/2014)...  Arena Pharmaceuticals, Inc. (NASDAQ: ARNA ) announced today ... and update at the Piper Jaffray 26 th Annual ... Eastern Time (5:30 a.m. Pacific Time), at The New York ... A live audio webcast of the presentation will be ... www.arenapharm.com . A replay of the presentation will be ...
(Date:11/24/2014)... , Nov. 24, 2014 /PRNewswire/ - Milestone Pharmaceuticals, ... trial conducted in Melbourne, Australia ... for the potential treatment of paroxysmal supraventricular tachycardia ... of MSP-2017 was well tolerated at single doses ... excellent safety profile, desirable PK properties including rapid ...
(Date:11/24/2014)... , Nov. 24, 2014  Marta Rendon, MD, founder ... Boca Raton , FL and international key opinion leader ... from the United States to attend ... Istanbul, Turkey . Here, at this gathering of ... as world renowned facial plastic surgery experts from ...
Breaking Medicine Technology:Arena Pharmaceuticals to Present at Piper Jaffray 26th Annual Healthcare Conference 2Arena Pharmaceuticals to Present at Piper Jaffray 26th Annual Healthcare Conference 3Milestone Announces Positive Phase 1 Data for MSP-2017; Supports Advancement into Phase 2 in PSVT Patients 2Milestone Announces Positive Phase 1 Data for MSP-2017; Supports Advancement into Phase 2 in PSVT Patients 3Marta Rendon, MD Selected To Attend Prestigious, Global Aesthetic Experts Summit! 2Marta Rendon, MD Selected To Attend Prestigious, Global Aesthetic Experts Summit! 3
... DIEGO, Nov. 18 Rules-Based Medicine, Inc. (RBM) today announced ... with Covance Inc. (NYSE: CVD ) that will ... biomarker technologies and services for use in drug development. Under ... exclusive third-party provider of multiplexed protein biomarker testing services, and ...
... , , TEMECULA, Calif., Nov. 17 ... announced third quarter 2009 results. , "We are continuing ... increases in our gross margins," stated Steven H. Kane, ... third quarter revenues from sales of safety sponges were ...
Cached Medicine Technology:Rules-Based Medicine Forms Biomarker Alliance With Covance Inc. 2Rules-Based Medicine Forms Biomarker Alliance With Covance Inc. 3Patient Safety Technologies Reports Third Quarter Results 2Patient Safety Technologies Reports Third Quarter Results 3Patient Safety Technologies Reports Third Quarter Results 4Patient Safety Technologies Reports Third Quarter Results 5Patient Safety Technologies Reports Third Quarter Results 6
(Date:11/26/2014)... OR (PRWEB) November 26, 2014 The ... fresh water shortages, is moving forward with a plan ... drinking water.* Radio host Sharon Kleyne supports the San ... “Total Water Management” (TWM). According to Kleyne, by recycling ... constantly import new water could be greatly reduced. (Spagat, ...
(Date:11/26/2014)... Loffler Companies has been selected as ... an online information and news publication for resellers of ... consecutive year Loffler has earned this prestigious award and ... Dealer Awards honor the best and the brightest in ... honorees were chosen for their innovative marketing and sales ...
(Date:11/26/2014)... WOODLAND HILLS, CA (PRWEB) November 26, 2014 ... implants costing thousands of dollars, the prosthodontists at the Anacapa ... that paying for dental procedures can be daunting for some. ... to pay for the procedures they offer. , “No dentist ... functioning and beautiful set of teeth,” said Dr. Mamaly Reshad, ...
(Date:11/26/2014)... Senior Planning Services, providing “expert guidance for ... providing the adult demographic education and access to ... the rough seas of applying for Medicaid. Part of ... topic of Medicaid eligibility and proper planning, while also ... for this coverage. , Senior Planning Services has ...
(Date:11/26/2014)... ISI Telemanagement Solutions, Inc., a ... award the 2014 Lync Pioneer Award ... as a great honor to ISI, who continues ... Specifically, ISI offers an integrated collaboration reporting and ... including voice, video, Lync IM, and conferencing. ...
Breaking Medicine News(10 mins):Health News:Global fresh water advocate supports San Diego wastewater recycling plan 2Health News:Global fresh water advocate supports San Diego wastewater recycling plan 3Health News:Loffler Companies Recognized as an Elite Dealer for Sixth Consecutive Year 2Health News:Anacapa Dental Art Institute Doctors Offers 6 Tips on How to Pay for Cosmetic, Restorative and Implant Dentistry 2Health News:Anacapa Dental Art Institute Doctors Offers 6 Tips on How to Pay for Cosmetic, Restorative and Implant Dentistry 3Health News:Anacapa Dental Art Institute Doctors Offers 6 Tips on How to Pay for Cosmetic, Restorative and Implant Dentistry 4Health News:Anacapa Dental Art Institute Doctors Offers 6 Tips on How to Pay for Cosmetic, Restorative and Implant Dentistry 5Health News:Senior Planning Services Teams Up with SeniorNet To Educate Seniors On Medicaid Planning 2Health News:ISI Telemanagement Solutions, Inc. has been Awarded the 2014 Microsoft Lync Pioneer Award 2
... French and Spanish . , Researchers from ... belonging to the Laboratorio de Ciencias de la Visin y Aplicaciones, ... the tool required to implement it, which has been named "halometer". ... and a computer where the mouse is used as a response ...
... the old journalistic adage goes. Not necessarily, say researchers from ... of the Jewish General Hospital. In a first-of-its kind study ... versus 20 years ago, positivity and hope seem to be ... potential impact on patient perspectives," said Dr. Melissa Henry, the ...
... Epidemiology Unit, Southampton, UK, and Dr. Michal Hiligsmann ... with the ESCEO Young Investigator Awards, supported by a ... European Congress on Osteoporosis & Osteoarthritis (ECCEO11-IOF) now taking ... 2,500, recognize young investigators for their excellence in bone ...
... March 23, 2011 Although acupuncture is commonly used ... remain. Investigators from the Universities of Exeter & Plymouth ... (Daejeon, South Korea) critically evaluated systematic reviews of acupuncture ... this question. Reporting in the April 2011 issue of ...
... with Sichuan University in Chengdu, China, investigated eye movements in ... mechanisms that control them and how they compare between different ... movement, that is rare in British people, is much more ... differences in brain function between different populations. ...
... By Amanda Gardner HealthDay Reporter , ... developed a new way of predicting when breast cancer ... "epigenetic signatures" seem to distinguish more aggressive cancers from ... Epigenetic alterations do not involve changes to the ...
Cached Medicine News:Health News:Researchers develop a halometer that tests alterations in night vision 2Health News:Researchers develop a halometer that tests alterations in night vision 3Health News:Good news! Hope makes headlines 2Health News:Acupuncture for pain no better than placebo and not without harm 2Health News:Eye movement differs in British and Chinese populations 2Health News:Genes' 'On/Off' Switches Yield Clues to Breast Cancer 2Health News:Genes' 'On/Off' Switches Yield Clues to Breast Cancer 3
... is effective against gram positive and gram ... interfering with bacterial protein synthesis by binding ... suggested working concentration of 50 mg/L is ... thereby making a gentler alternative to penicillin ...
... new approach to practice management, built to address ... healthcare market. OPIE was created by practicing O&P ... in this field, so it thinks the way ... than anything else available. If you believe there ...
... new hand-held Slit Lamp HSL150 with an ... For Human and Veterinary medicine for the ... an ergonomic, compact,design, the new HEINE HSL ... slit lamp is not available,or practical to ...
... used for endophotocoagulation indications, ... uncompromised laser treatment of ... handpieces are compatible with ... (532 nm), OcuLight infrared ...
Medicine Products: