Horizon Pharma stops development of Actimmune for Friedreich’s ataxia

FA is a life-shortening and degenerative neuro-muscular disorder that affects nearly 4,000 to 6,000 people in the US.

The placebo-controlled trial, Steadfast, failed to demonstrate that Actimmune therapy resulted in statistically significant changes from baseline in the modified Friedreich’s ataxia rating scale (FARS-mNeuro), at 26 weeks.

Apart from this, the secondary endpoints did not meet statistical significance.

Horizon Pharma is also stopping the 26-week extension study and the long-term safety study.

The company said will continue to work with the Friedreich's Ataxia Research Alliance (FARA) and the principal investigator to further assess the data for future research efforts as well as data presentation or publication.

Horizon Pharma chairman, president and CEO Timothy Walbert said: "A well-designed, rigorous study like Steadfast would not have been possible without the extraordinary drive of the FA community, particularly the people who enrolled in the study, the clinical trial investigators and the Friedreich's Ataxia Research Alliance. 

"While the results were not what we hoped for, this is the very reason why research and development is important – to find answers that may help inform future research."

Actimmume is a biologically manufactured protein similar to one the body makes naturally to help avoid infection.  

The US Food and Drug Administration approved tt for use in two rare diseases.  It is indicated to reduce the frequency and severity of serious infections associated with Chronic Granulomatous Disease and for delaying time to disease progression for patients with severe, malignant osteopetrosis.

Image: Horizon Pharma stops development of Actimmune for Friedreich's ataxia. Photo: courtesy of jk1991 at FreeDigitalPhotos.net.