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news.Horizon Pharma has completed target enrollment of its Phase 3 study evaluating ACTIMMUNE (interferon gamma-1b) for the treatment of people with Friedreich's ataxia (FA), a degenerative neuro-muscular disorder.
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The study (NCT02415127) has reached its target enrollment of 90 patients at four sites in the United States, and top-line results are expected by the end of 2016.
"The achievement of this important milestone would not have been possible without the passionate commitment of people living with FA, the Friedreich’s Ataxia Research Alliance and our Phase 3 study investigators," said Jeffrey W. Sherman, M.D., FACP, executive vice president, research and development and chief medical officer, Horizon Pharma plc.
"We are grateful for their collective partnership and guidance, which drives our efforts toward providing a potential treatment option for this debilitating disorder."
The Safety, Tolerability and Efficacy of ACTIMMUNE Dose Escalation in Friedreich’s Ataxia study ("STEADFAST") is a randomized, multi-center, double-blind, placebo-controlled study with patients randomized 1:1 to receive subcutaneous doses of either ACTIMMUNE or placebo three times a week for a total of 26 weeks.
The primary endpoint will 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. In addition to safety and efficacy, the STEADFAST trial will evaluate the pharmacokinetic characteristics of ACTIMMUNE in people with FA.
After completion of the study, patients who participated in STEADFAST will have the opportunity to transition to an open-label extension study (NCT02593773).
"The prompt recruitment and enrollment of this study reflects the urgent unmet need as well as the commitment to participate in research among people living with FA," said Ronald J. Bartek, co-founder and founding president, Friedreich’s Ataxia Research Alliance (FARA).
"As an organization dedicated to the pursuit of scientific research leading to treatments for FA, we are very pleased with the progress of the STEADFAST study and are hopeful that the results lead to the first approved treatment for people living with FA."
In April 2015, ACTIMMUNE was granted Fast Track status for FA by the U.S. Food and Drug Administration (FDA). This designation provides greater access to and more frequent communication with the FDA throughout the entire drug development and review process, with the goal of possibly expediting approval.
Fast Track designation also gives Horizon Pharma the opportunity to potentially submit sections of the ACTIMMUNE registration dossier for FA on a rolling basis, and allows ACTIMMUNE to be considered for priority review at the time of submission based on forthcoming clinical data.
About Friedreich’s ataxia (FA)
FA is a debilitating, life-shortening and degenerative neuro-muscular disorder that affects approximately 4,000 people in the United States. Onset of symptoms can vary from five years old to adulthood, with the childhood onset tending to be associated with a more rapid progression. A progressive loss of coordination and muscle strength leads to motor incapacitation and often the full-time use of a wheelchair. Most young people diagnosed with FA require mobility aids such as a cane, walker or wheelchair by their teens or early 20’s. There are currently no approved treatments for FA.
About ACTIMMUNE
ACTIMMUNE (interferon gamma-1b) is a biologically manufactured protein similar to one the body makes naturally to help prevent infection. ACTIMMUNE is currently approved by the U.S. Food and Drug Administration (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 cells of the immune system. In addition, ACTIMMUNE is indicated to slow the worsening of severe, malignant osteopetrosis (SMO), a genetic disorder that affects normal bone formation.