Re: Farmas USA
Horizon Pharma plc (NASDAQ:HZNP) submits an Investigational New Drug (IND) application to the FDA for Actimmune (interferon gamma-1b) for the expanded indication of the treatment of Friedreich's ataxia (FA). It also requests Fast Track designation.
The company plans to start a Phase 3 clinical trial in Q2 in collaboration with the Friedreich's Ataxia Research Alliance.
The primary efficacy endpoint of the 90-subject, randomized, placebo-controlled, study is the change in Friedreich's Ataxia Rating Scale-modified neurological exam score from baseline to 26 weeks versus placebo. A six-month open-label extension study will be offered to the FA patients who complete the trial.
Actimmune is currently approved for reducing the frequency and severity of serious infections associated with chronic granulomatous disease and the delaying time to disease progression in patients with severe malignant osteopetrosis.
HZNP