Horizon Pharma plc Initiates Phase 3 Trial of ACTIMMUNE(R) (interferon gamma-1b) for the Treatment of People With Friedreich's Ataxia - Seite 2

In April 2015, ACTIMMUNE was granted Fast Track status for FA by the U.S. Food and Drug Administration (FDA). This designation provides greater access to and more frequent communication with the FDA throughout the entire drug development and review process, with the goal of possibly expediting approval. Fast Track designation also gives Horizon Pharma the opportunity to potentially submit sections of the ACTIMMUNE registration dossier for FA on a rolling basis, and allows ACTIMMUNE to be considered for priority review at the time of submission based on forthcoming clinical data.

About Friedreich's ataxia (FA)
FA is a debilitating, life-shortening and degenerative neuro-muscular disorder that affects approximately 4,000 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. There are currently no approved treatments for FA. 

About FARA
The Friedreich's Ataxia Research Alliance (FARA) is a national, public, 501(c)(3), non-profit, tax exempt organization dedicated to curing Friedreich's ataxia (FA), a rare neuromuscular disorder, through research. For more information about FA, visit the Friedreich's Ataxia Research Alliance (FARA) website at www.curefa.org.

About ACTIMMUNE®
ACTIMMUNE (interferon gamma-1b) is a biologically manufactured protein similar to one the body makes naturally to help prevent infection. ACTIMMUNE is currently approved by the U.S. Food and Drug Administration (FDA) for use in two rare diseases. It is indicated to reduce the frequency and severity of serious infections associated with Chronic Granulomatous Disease (CGD), a genetic disorder that affects the functioning of some cells of the immune system. In addition, ACTIMMUNE is indicated to slow the worsening of severe, malignant osteopetrosis (SMO), a genetic disorder that affects normal bone formation. For more information, please see www.ACTIMMUNE.com.