Orphan status for Horizon Pharma's Actimmune in Friedreich's ataxia
The US Food and Drug Administration has granted orphan drug designation for Ireland-headquartered specialty biopharma firm Horizon Pharma’s (Nasdaq: HZNP) Actimmune (interferon gamma-1b) for the treatment of Friedreich's ataxia (FA), a rare, genetic disease for which there are currently no approved treatments.
Actimmune became part of Horizon’s portfolio with the around $660 million acquisition earlier this year of privately-held Irish drugmaker Vidara Therapeutics International, a tax inversion deal that resulted in the US company moving its tax base to Ireland.
Actimmune is already approved for reducing the frequency and severity of serious infections associated with chronic granulomatous disease. It is also indicated for delaying time to disease progression in patients with severe, malignant osteopetrosis.
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