Horizon Pharma plcHZNP announced that the FDA has granted fast track status to Actimmune for the treatment of Friedreich's ataxia. Actimmune is already approved in the U.S. for the treatment of patients suffering from two rare diseases - chronic granulomatous disease and severe, malignant osteopetrosis.
Currently, Horizon Pharma is working toward expanding the product's label in additional indications.
Fast track designation is granted to candidates that are being developed for the treatment of a serious or life-threatening disease or condition and is believed to possess the capability to address unmet medical needs. On receiving this designation, Horizon Pharma may communicate with the FDA frequently throughout the entire development and review process, with the aim of expediting the candidate's approval. Moreover, the company is also eligible to make a rolling submission of new drug application for the candidate, which will be considered for priority review.
Horizon Pharma intends to commence a randomized, double-blind, multi-center, placebo-controlled phase III study on Actimmune in the second quarter of 2015 for the treatment of Friedreich's ataxia in patients aged between 10 and 25 years. The primary endpoint for the study will measure the change from baseline in the patients after 26 weeks of treatment with ACTIMMUNE compared to placebo. The study will be conducted in collaboration with the Friedreich's Ataxia Research Alliance (FARA) and a sub-set of FARA's Collaborative Clinical Research Network study centers. Actimmune enjoys orphan drug designation in the U.S. for Friedreich's ataxia.
According to the company, there are no therapies approved by the FDA for the treatment of Friedreich's ataxia. Given that one in 50,000 people in the U.S. suffer from the disease, there is strong unmet need for drugs in the treatment of Friedreich's ataxia.
Horizon Pharma carries a Zacks Rank #1 (Strong Buy). Other well-ranked stocks in the health care sector include ANI Pharmaceuticals, Inc. ANIP , Cytokinetics, Incorporated CYTK and Acorda Therapeutics, Inc. ACOR . All these stocks carry the same rank as Horizon Pharma.