Horizon Pharma plc Receives Orphan-Drug Designation for ACTIMMUNE® (Interferon gamma-1b) in Friedreich's Ataxia

DUBLIN, IRELAND–(Marketwired - Oct 3, 2014) - Horizon Pharma plc (NASDAQ: HZNP), a specialty biopharmaceutical company with a portfolio of products in arthritis, inflammation and orphan diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for ACTIMMUNE® (Interferon gamma-1b) for the treatment of Friedreich’s ataxia (FA), a rare, genetic disease for which there are currently no approved treatments.
The U.S. Orphan Drug Act (ODA) 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 FA, with seven years of market exclusivity for this indication.
FA is a debilitating, life-shortening, degenerative neuro-muscular disorder that affects about one in 50,000 people in the United States. Onset of symptoms can vary from five years old to adulthood, with the childhood onset tending to be associated with a more rapid progression. A progressive loss of coordination and muscle strength leads to motor incapacitation and often the full-time use of a wheelchair. Most young people diagnosed with FA require mobility aids such as a cane, walker or wheelchair by their teens or early 20’s.
“This orphan-drug designation for ACTIMMUNE is an important regulatory advancement as we build our development program in FA, a rare, debilitating disorder with no currently approved treatments,” said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. "We look forward to the release of results from a Phase 2 study sponsored by the Friedreich’s Ataxia Research Alliance (FARA) and conducted at Children’s Hospital of Philadelphia."
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 (CGD), 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 (SMO), 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. For more information, please visit www.ACTIMMUNE.com.

Sorry Alan at times I am not the brightest guy you will ever talk to, but is this article saying that Actimmune has shown to be beneficial to patients with FA. And with it being given orphan status[ because it is for a rare disease] it is an incentive for pharmaceuticals to produce it.

I wonder if any of the SCA,s have been considered for orphan status?

Many thanks again Alan for your tireless work.