Fast-track for Edison’s ataxia drug

A new treatment for the rare disease Friedreich’s ataxia has been given ‘fast track’ status by the FDA.

Edison’s EPI-743 is being developed for a range of paediatric and adult mitochondrial disease, of which Friedreich’s ataxia is just one.

If Edison can gain approval, EPI-743 will be the first ever treatment for Friedreich’s ataxia, and already has orphan status in the US for the condition.

Different types of mitochondrial disease all share common biochemical defects in the mitochondrion, special ‘power generators’ found in the body’s cells.

Friedreich’s ataxia is an autosomal recessive nuclear DNA inherited mitochondrial disease, and affects an estimated 1 in 50,000 individuals in the United States and Europe. Friedreich’s ataxia is caused by a defect in the gene frataxin. Patients have “energy failure” symptoms, including ataxia, muscle weakness, heart failure, diabetes, and visual, speech and hearing deficiencies. Friedreich’s ataxia is a highly debilitating and life-shortening disease.

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 trial in adults with Friedreich’s ataxia is fully enrolled and is expected to be completed in the third quarter of 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.

“Fast Track designation will facilitate Edison’s clinical development of EPI-743 for patients with Friedreich’s ataxia,” said Guy Miller, MD, PhD, chairman and chief executive 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 programme 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. The system 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 programme,” stated Mr. 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.”

Pearson Syndrome

Edison has also just announced the launch of a phase 2 study of EPI-743 (Vincerinone) in children with Pearson Syndrome.

The condition is an ultra-rare, fatal mitochondrial disease characterised by sideroblastic anemia, pancytopenia and pancreatic dysfunction. There is no treatment for Pearson syndrome and most patients die in infancy or early childhood because of metabolic disorders or infections caused by the condition.

Rare diseases – where are the patients?



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