Horizon Pharma Begins Phase 3 Trial of ACTIMMUNE For The Treatment of Patients With Friedreich’s Ataxia

Horizon Pharma has recently announced it initiated a Phase 3 study evaluating the Safety, Tolerability and Efficacy of ACTIMMUNE Dose Escalation in Friedreich’s Ataxia study (“STEADFAST”) of ACTIMMUNE (interferon gamma-1b) for the treatment of people with Friedreich’s Ataxia (FA).

ACTIMMUNE (Interferon gamma 1-b) is a biologically manufactured protein similar to a protein naturally produced by the body. “As a rare disease with no currently approved treatments, Friedreich’s Ataxia represents an area of significant unmet medical need,” said in a recent press release Timothy P. Walbert, Chairman, President and Chief Executive Officer, Horizon Pharma plc. “The Phase 3 study represents a significant next step in evaluating ACTIMMUNE in this population, which is approximately four thousand people in the United States. We look forward to continuing to work with the FDA with the goal of providing a potential treatment option to the FA community.”

The trial, which intends to recruit 90 FA patients at four different locations across the U.S., is a multi-center, double-blind, randomised, placebo-controlled study where patients will be randomly assigned to receive either ACTIMMUNE or a placebo, three times per week for a total of 26 weeks. The study’s primary endpoint are the changes in neurological outcomes, assessed with a modified version of the Friedreich’s Ataxia Rating Scale (mFARS), measuring neurological outcomes in the upper and lower extremity coordination improvement from baseline. Higher scores in FARS reflect greater disability levels. The trial will also assess the pharmacokinetic features of the compound in FA patients.

“This clinical trial is an example of the progress the scientific community has made in understanding FA,” added  Ronald J. Bartek, president, director and co-founder, Friedreich’s Ataxia Research Alliance (FARA). “Less than 20 years ago, we had not identified the cause of FA, and today we have a late-stage clinical study that brings us one step closer to potentially having the first treatment for people with FA.”

Last April the FDA had granted ACTIMMUNE a Fast Track status for FA, a designation that provides access and frequent communication with the FDA concerning drug development and review, potentiating faster drug approval.