Horizon Pharma files IND application for Actimmune to treat Friedreich’s Ataxia

Simultaneously, the company has requested the FDA for fast track designation for Actimmune.

The company also intends to commence a Phase III trial in the second quarter of 2015 in collaboration with the Friedreich’s Ataxia Research Alliance (FARA) and the investigators and clinics of FARA’s Collaborative Clinical Research Network (CCRN) in FA.

The Phase III trial is designed to evaluate Actimmune in around 90 male and non-pregnant female subjects between the ages of 10 and 25 years, inclusive, with an FA functional stage of greater than 1 to less than 5 and the ability to walk 25 feet with or without an assistive device.

Horizon Pharma chairman, president and chief executive officer Timothy Walbert said: "This is a significant step forward in the further development of Actimmune beyond the approved indications of chronic granulomatous disease and severe, malignant osteopetrosis.

"We have established a successful partnership with the Friedreich’s Ataxia Research Alliance and its Collaborative Clinical Research Network in Friedreich’s Ataxia and look forward to working with them on this study in order to potentially bring a treatment option to the estimated 3,700 people in the United States with this devastating disease."

During the randomized, double-blind, multicenter, placebo-controlled, 26-week Phase III trial, subjects will be randomized 1:1 to receive either Actimmune or matching placebo.

The trial’s primary endpoint will be the change in the Friedreich’s Ataxia Rating Scale-modified neurological exam score (FARS-mNeuro), which is a measure of disease activity and correlates significantly with functional disability, from baseline to 26 weeks for patients treated with Actimmune compared to placebo.

Patient enrolment in the trial is expected to take 18 months for completion.

The company said that a six-month open-label extension trial will be offered to those FA patients completing the Phase III trial.