Edison's vatiquinone receives Orphan Status for treatment of Friedreich's ataxia

Edison Pharmaceuticals today announced that the US Food and Drug Administration has granted Orphan Status to vatiquinone for the treatment of Friedreich's ataxia.

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. It is used to identify the active pharmacological ingredient in a drug and is also known as the generic name.

EPI-743 is currently in phase 2 development for the treatment of Friedreich's ataxia. A phase 2B randomized double-blind, placebo-controlled trial in adults with Friedreich's ataxia is fully enrolled at this time. A study in patients with the rare point mutation genotype is actively enrolling subjects. Details of these trials can be found on clinicaltrials.gov. 

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, notably a more expedited drug approval process and an extended period of market exclusivity.

The FDA has previously granted orphan status to EPI-743 for the treatment of inherited respiratory chain diseases. In addition, EPI-743 has received orphan designation for Leigh syndrome by the Committee on Orphan Medicinal Products (COMP) of the European Medicines Agency.

Source:

Edison Pharmaceuticals, Inc.

Comments

The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
Post a new comment
Post

While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. We do not provide medical advice, if you search for medical information you must always consult a medical professional before acting on any information provided.

Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles.

Please do not ask questions that use sensitive or confidential information.

Read the full Terms & Conditions.

You might also like...
Semaglutide shows no higher 12-month risk of adverse neuropsychiatric outcomes than other antidiabetic drugs, study suggests