FDA Grants Edison Pharmaceuticals' EPI-743 Orphan Status for Leigh Syndrome

 FDA Grants Edison Pharmaceuticals' EPI-743 Orphan Status for Leigh Syndrome

EPI-743 in phase 2B development for Leigh syndrome

PR Newswire

MOUNTAIN VIEW, Calif., June 9, 2014

MOUNTAIN VIEW, Calif., June 9, 2014 /PRNewswire/ --Edison Pharmaceuticals
today announced that the FDA has granted Orphan Drug status to vatiquinone for
the treatment of Leigh syndrome.

Vatiquinone is the International Nonproprietary Name (INN) for Edison's
EPI-743. The INN is a unique international name issued by the World Health
Organization, which is used to identify the active pharmacological ingredient
in a drug. This is also known as the generic name.

EPI-743 is currently in phase 2B/3 development for the treatment of Leigh
syndrome. A phase 2B randomized double-blind placebo-controlled trial in
children with Leigh syndrome is fully enrolled in the United States, and a
phase 2B/3 trial of EPI-743, which is being conducted in conjunction with
Dainippon Sumitomo Pharma Co, Ltd, is ongoing in Japan.

Orphan designation was established as part of the Orphan Drug Act which was
passed by the US Congress in 1983 to encourage the development of drugs for
the treatment of rare, orphan diseases. The FDA grants orphan status to drugs
that are being developed specifically to treat a rare condition and have shown
potential benefit for the indication. Orphan designation affords several
advantages including a more expedited drug approval process and an extended
period of market exclusivity.

Edison Pharmaceuticals has previously received Orphan Designation from the FDA
for the treatment of inherited respiratory chain diseases of the mitochondria
and Friedreich's ataxia, and from the European Medicines Agency Committee on
Orphan Products and Japanese Ministry of Health, Labor and Welfare for the
treatment of Leigh syndrome.

Leigh Syndrome
Leigh syndrome is an inherited lethal, progressive, predominately pediatric,
neuromuscular disorder for which there are no approved treatments. It is the
most common pediatric inherited mitochondrial disease. Initially described in
1951, the hallmarks of the disease include bilateral necrosis (death) of
central nervous system regions responsible for the control of breathing and
other neurologic functions. Leigh syndrome belongs to a large family of
disorders identified as "mitochondrial disease." The disorders share as a
common biochemical feature defects in cellular energy metabolism.

EPI-743 is an orally bioavailable small molecule being developed by Edison
Pharmaceuticals for inherited mitochondrial diseases, and is a member of the
para-benzoquinone class of drugs. The mechanism of action of EPI-743 involves:
i) augmenting the synthesis of glutathione; ii) optimizing metabolic control;
iii) enhancing the expression of genetic elements critical for cellular
management of oxidative stress; and iv) acting at the mitochondria to regulate
electron transport. The utility of EPI-743 is also being explored in other
inherited respiratory chain disorders.

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

SOURCE Edison Pharmaceuticals, Inc.

Website: http://www.edisonpharma.com
Contact: Edison Information, Edison Pharmaceuticals, Inc., 350 North Bernardo
Avenue, Mountain View, CA 94043, info@edisonpharma.com, edisonpharma.com
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