Drug Discovery and Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • R&D 100 Awards
  • Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50

FDA Fast Tracks Edison’s New Friedreich’s Ataxia Drug

By Drug Discovery Trends Editor | March 17, 2014

Edison Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to EPI-743, the company’s lead drug, for the treatment of Friedreich’s ataxia. EPI-743 is being developed for pediatric and adult mitochondrial disease, including Friedreich’s ataxia. 
 
EPI-743 has successfully completed Phase 1 and multiple Phase 2 studies. In these studies, EPI-743 has been demonstrated to be safe and well tolerated. Two Phase 2 trials in patients with Friedreich’s ataxia are ongoing. A Phase 2B randomized double-blind placebo-controlled trial in adults with Friedreich’s ataxia is fully enrolled. It is expected to be completed in Q3, 2014. In addition, EPI-743 is being evaluated in a rare Friedreich’s ataxia genetic subtype- patients with a point mutation in the gene encoding frataxin. This single-arm subject-controlled trial is also fully enrolled. Details of these trials can be found on clinicaltrials.gov. 
 
“Fast Track designation will facilitate Edison’s clinical development of EPI-743 for patients with Friedreich’s ataxia,” said Guy Miller, chairman and CEO of Edison Pharmaceuticals. “We are fully committed to delivering the first approved drug for this highly debilitating and lethal disease for which there are no FDA-approved therapies.”
 
The FDA’s Fast Track program is designed to facilitate the development of drugs that have demonstrated potential to treat diseases that are serious, life threatening, and for which there is an unmet medical need. Fast Track provides a number of benefits, including the ability to meet and communicate more frequently with the FDA to discuss drug development plans, as well as eligibility for accelerated approval. Drugs with Fast Track designation may also receive “rolling review” from the FDA. This permits a drug company to submit completed portions of a New Drug Application (NDA) for immediate review before the entire application is completed. 
 
“We greet with tremendous enthusiasm the FDA’s issuance of Fast Track designation to EPI-743 for Edison’s Friedreich’s ataxia development program,” said Ron Bartek, president, Friedreich’s Ataxia Research Alliance (FARA) and chairman of the board of the National Organization for Rare Disorders. “This demonstrates the FDA’s commitment to the accelerated development of promising drugs for diseases which have no treatments.”
 
The FDA has previously granted Orphan designation status to EPI-743 for the treatment of Friedreich’s ataxia.
 
Date: March 17, 2014
Source: Edison Pharmaceuticals
 

Filed Under: Drug Discovery

 

Related Articles Read More >

Takeda Pharmaceutical in the Drug Discovery & Development Pharma 50
Takeda’s Takhzyro fares well in pediatric hereditary angioedema study
Novartis in the Pharma 50
Novartis to cut up to 8,000 positions
Gilead Sciences in the Drug Discovery & Development Pharma 50
Gilead resubmits application to FDA for twice-yearly HIV drug lenacapavir
George Floyd mural
How the pandemic and George Floyd made clinical trial diversity a priority

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.

Need Drug Discovery news in a minute?

We Deliver!
Drug Discovery & Development Enewsletters get you caught up on all the mission critical news you need. Sign up today.
Enews Signup
Drug Discovery and Development
  • MASSDEVICE
  • DEVICETALKS
  • Medical Design & Outsourcing
  • MEDICAL TUBING + EXTRUSION
  • MEDTECH 100
  • Medical Design Sourcing
  • Subscribe to our Free E-Newsletter
  • Contact Us
  • About Us
  • Advertise With Us
  • R&D World
  • Drug Delivery Business News
  • Pharmaceutical Processing World

Copyright © 2022 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search Drug Discovery & Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • R&D 100 Awards
  • Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50