Horizon Pharma’s Friedreich’s Ataxia Drug Gets Fast-Track Designation

April 30, 2015

Horizon Pharma PLC said Friday that its experimental Friedreich’s ataxia treatment has received fast-track designation from the U.S. Food and Drug Administration, a move that could speed approval.
The Dublin-based specialty pharmaceutical company said the drug, Actimmune, is a treatment for Friedreich’s ataxia, an inherited disease causing nervous-system damage that often results in motor incapacitation and the use of a wheelchair.

Horizon submitted an investigational new drug application, which requests FDA authorization to test on human subjects, in February and has said it plans to begin a late-stage trial in the second quarter.

Horizon said there are currently no FDA-approved treatments for the disorder.

The FDA assigns fast-track status to drugs and vaccines that are meant to address serious conditions and unmet medical needs with the goal of expediting approval.

Actimmune is already approved by the FDA for use in two genetic diseases.


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