Horizon Pharma Gets Orphan-Drug Designation For Actimmune In Friedreich's Ataxia

(RTTNews) - Horizon Pharma plc (HZNP) said Friday that the U.S. Food and Drug Administration has granted orphan-drug designation for Actimmune for the treatment of Friedreich's ataxia, a rare, genetic disease for which there are currently no approved treatments.

The U.S. Orphan Drug Act provides for granting special status to a drug or biological product to treat a rare disease or condition that affects fewer than 200,000 patients in the United States. Orphan designation qualifies the sponsor of the drug for various development incentives, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received orphan-drug designation is not subject to the Prescription Drug User Fee Act (PDUFA) fees.

The designation may provide Actimmune, if approved for the treatment of Friedreich's ataxia, with seven years of market exclusivity for this indication.

Actimmune is currently approved for two rare diseases in the United States. It is approved by the FDA to reduce the frequency and severity of serious infections associated with Chronic Granulomatous Disease, a genetic disorder that affects the functioning of a type of white blood cell of the immune system, neutrophils or phagocytes, leading to recurrent severe bacterial and fungal infections and chronic inflammatory conditions. In addition, Actimmune is approved by the FDA to slow the worsening of severe, malignant osteopetrosis, a genetic disorder, that affects normal bone formation causing the abnormal accumulation of bone material which tends to narrow the space inside bones where bone marrow is formed. This can cause failure of the bone marrow, leading to a decrease in various blood cells such as red blood cells and white blood cells.