US FDA grants Edison Pharmaceuticals' EPI-743 orphan status for Leigh Syndrome

9 June 2014

The US Food and Drug Administration has granted orphan drug status to privately-held US pharma company Edison Pharmaceuticals’ vatiquinone for the treatment of Leigh syndrome.

In February, the FDA also granted the drug orphan status for the treatment of Friedreich's ataxia. Vatiquinone is the generic name for Edison's EPI-743 which is currently in Phase IIb/III development for the treatment of Leigh syndrome, an inherited lethal, progressive, predominately pediatric, neuromuscular disorder for which there are no approved treatments. It is the most common pediatric inherited mitochondrial disease and belongs to a large family of disorders identified as “mitochondrial disease.”

Ongoing trials

This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.

Try before you buy

Free

7 day trial access

Take a Free Trial

All the news that moves the needle in pharma and biotech
Exclusive features, podcasts, interviews, data analyses and commentary from our global network of life sciences reporters.
Receive The Pharma Letter daily news bulletin, free forever.

Become a subscriber

£820

Or £77 per month

Subscribe Now

Unfettered access to industry-leading news, commentary and analysis in pharma and biotech.
Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results.
Daily roundup of key events in pharma and biotech.
Monthly in-depth briefings on Boardroom appointments and M&A news.
Choose from a cost-effective annual package or a flexible monthly subscription

The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed

Chairman, Sanofi Aventis UK