Ireland-based Horizon Pharma has started the Phase III study of Actimmune (interferon gamma-1b) to treat patients with Friedreich’s Ataxia (FA), a degenerative neuro-muscular disorder.
The Phase III safety, tolerability and efficacy of Actimmune dose escalation in Friedreich’s Ataxia study (STEADFAST) is a randomised, multi-centre, double-blind and placebo controlled trial with patients randomised 1:1 to receive subcutaneous doses of either Actimmue or placebo three times a week for a total of 26 weeks.
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Horizon Pharma president and CEO Timothy Walbert said: "The Phase III study represents a significant next step in evaluating Actimmune in this population, which is approximately four thousand people in the US.
"We look forward to continuing to work with the FDA with the goal of providing a potential treatment option to the FA community."
The company plans to enrol around 90 patients in the trial at four sites in the US.
According to the firm, the primary endpoint will assess the change in neurological outcome and evaluate the effect of actimmune against placebo as measured by the modified Friedreich’s Ataxia Rating Scale (mFARS), focused on objective neurologic measures such as upper and lower extremity coordination improvement from baseline.
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By GlobalDataThe study will assess the pharmacokinetic characteristics of Actimmune in people with FA, in addition to safety and efficacy.
In April, the company received fast track status from the US Food and Drug Administration (FDA) for Actimmune for FA.
