Thursday, September 13, 2012

EPI-743 now Orphan Drug!

EMA Grants Orphan Designation to Edison Pharmaceuticals for EPI-743 for Treatment of Leigh Syndrome

Clinical development of EPI-743 underway in Europe & the United States Mountain View, California; September 12, 2012. 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. Contact Information Edison Pharmaceuticals, Inc.
350 North Bernardo Avenue
Mountain View, CA 94043

Getting one step closer to making this drug available for all who suffer from Leigh's. It's a slow moving ship and I fear it won't get to market in time for many Mito friends. I thank God every day that we are able to be part of this drug trial. There are people like Dr. Gregg Enns who work tirelessly to help push research and clinical trials; he and others like him arereal world heros in my book.

You can help support him and others who are turning research and theory into meaningful and positive clinical trials by supporting the United Mitochondrial Foundation, They help fund doctors and researchers like Dr. Enns in the hopes of one day finding a cure.

Here is a link to UMDF's funded projects list:

My little super star!

Another option you could link / post on Facebook!

Nicole Schaper


  1. what a great picture! ahhhh. too cute! and what wonderful news about EPI getting orphan status. hugs! XOXO B

  2. It sounds like promising news. Thank God.

    I so hope things progress and will soon help many mito patients!!

  3. Good news indeed!! Now if we could only get Jagger on the drug!!!