Horizon gets FDA orphan drug status for ACTIMMUNE to treat Friedreich’s Ataxia

Currently, there are no approved treatments for FA, which is a debilitating, life-shortening, degenerative neuro-muscular disorder affecting at least one in 50,000 people in the US.

Most youngsters aged between 13 and 20 years, who are diagnosed with FA need mobility aids such as a cane, walker or wheelchair.

The orphan drug status might provide ACTIMMUNE, if approved to treat FA, with seven years of market exclusivity for this indication.

Horizon Pharma chairman, president and chief executive officer Timothy Walbert said the orphan-drug status for ACTIMMUNE is an important regulatory advancement as the company builds its development program in FA, a rare, debilitating disorder with no currently approved treatments.

"We look forward to the release of results from a Phase II study sponsored by the Friedreich’s Ataxia Research Alliance (FARA) and conducted at Children’s Hospital of Philadelphia," Walbert said.

In the US, ACTIMMUNE is currently approved to reduce the frequency and severity of serious infections associated with Chronic Granulomatous Disease (CGD) as well as to slow the worsening of severe, malignant osteopetrosis (SMO), a genetic disorder.

CGD is 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.

SMO affects normal bone formation causing the abnormal accumulation of bone material which tends to narrow the space inside bones where bone marrow is formed.

Image: The FDA orphan drug status may provide ACTIMMUNE seven years of market exclusivity for the treatment of FA. Photo: courtesy of Baitong333/ freedigitalphotos.net.