Ireland-based biopharmaceutical company Horizon Pharma has completed its target enrolment of 90 patients for Phase lll trial of actimmune (interferon gamma-1b) to treat Friedreich’s Ataxia (FA) disease.
FA is a degenerative neuro-muscular disorder that affects around 4,000 people in the US. There are currently no approved treatments for FA.
The study, titled Safety, Tolerability and Efficacy of ACTIMMUNE Dose Escalation in Friedreich’s Ataxia study (Steadfast), will be conducted at four sites in the US, with results expected by the end of this year.
Steadfast is a randomised, multi-centre, double-blind, placebo-controlled study with patients randomised 1:1 to receive subcutaneous doses of either actimmune or placebo three times a week for a total of 26 weeks.
The study’s primary endpoint is to evaluate the effect of actimmune versus placebo on the change from baseline to week 26 in neurological outcome, as measured by a modified version of the Friedreich’s Ataxia Rating Scale (FARS).
The FARS is used to measure neurological signs associated with FA, with higher scores reflecting a greater level of disability.
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Additionally, Steadfast will evaluate the pharmacokinetic characteristics of actimmune in people with FA.
After completion of the study, patients who participated in Steadfast will be transitioned to an open-label extension study.
In April last year, actimmune, a biologically manufactured protein, was granted fast track status for FA by the US Food and Drug Administration (FDA).
Actimmune is currently approved by the 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 immune system cells.
In addition, actimmune is indicated to decrease the worsening of severe, malignant osteopetrosis (SMO), a genetic disorder that affects normal bone formation.