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Horizon Pharma (HNZP) Starts Phase III Study on Actimmune - Analyst Blog

Horizon Pharma HZNP announced that it has initiated STEADFAST (safety, tolerability and efficacy of Actimmune dose escalation in friedreich's ataxia study), a phase III study, on Actimmune for the treatment of patients suffering from Friedreich’s ataxia.

The randomized, multi-center, double-blind, placebo-controlled study will enroll approximately 90 patients in the U.S. The primary endpoint for the study will measure the change in neurological outcome and evaluate the effect of Actimmune versus placebo in these patients.

We remind investors that in Apr 2015, the FDA had granted Fast Track status to Actimmune for the treatment of Friedreich’s ataxia. The company will be eligible to make a rolling submission of the new drug application for Actimmune, which will be considered for priority review.

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.

According to the company, there are no therapies approved by the FDA for the treatment of Friedreich’s ataxia, which affects approximately 4,000 people in the U.S. Thus, there is an unmet need for drugs addressing Friedreich’s ataxia.

Horizon Pharma carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector are Gilead Sciences Inc. GILD, Actelion Ltd. ALIOF and Hospira Inc. HSP. All three hold a Zacks Rank #1 (Strong Buy).

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HOSPIRA INC (HSP): Free Stock Analysis Report
 
GILEAD SCIENCES (GILD): Free Stock Analysis Report
 
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HORIZON PHARMA (HZNP): Free Stock Analysis Report
 
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