FDA Approves Primary Endpoint for Horizon’s FA Therapy Trial

Specialty biopharmaceutical company Horizon Pharma plc has released a business update on its primary care and orphan divisions, announcing that the U.S. Food and Drug Administration (FDA) has agreed on the primary endpoint for the phase 3 study dedicated to a potential Friedreich’s Ataxia (FA), called ACTIMMUNE. The company is planning to start enrolling pediatric patients for the study later this year.

The FDA has approved the alteration from baseline after 26 weeks in the Friedreich’s Ataxia Rating Scale-modified neurological exam score (FARS-mNeuro) for patients treated with ACTIMMUNE compared to placebo, as a primary endpoint for the phase 3 study to be conducted by Horizon to assess ACTIMMUNE. It is going to be carried out in partnership with the Friedreich’s Ataxia Research Alliance (FARA) and researchers of FARA’s Collaborative Clinical Research Network (CCRN) in Friedreich’s Ataxia.

The randomized, double-blind, multicenter, placebo-controlled trial, which is scheduled to start enrolling patients in the second quarter of 2015, is planned to include 110 participants, between the ages of 10 and 25 years old, who suffer from FA functional stage > 1 (minimal disability) to < 5 (severe disability). The patients will be administered either ACTIMMUNE or placebo at one of the four U.S. study centers.

The study is planned to be completed in 18 months, and the company has also announced that it is going to submit an Investigational New Drug (IND) application during the next three months. The drug has already been evaluated in a phase 3 trial, and the results of the FARS-mNeuro score suggested consistency with previous findings from the total FARS score in the study. The FARS-mNeuro is a subscore of the total FARS score removing components of the total FARS score viewed by FDA to be more subjective and effort dependent.

ACTIMMUNE has already been approved by the FDA to be used in the U.S. for the decrease of both the frequency and severity of serious infections in patients who suffer from Chronic Granulomatous Disease (CGD), or to delay the worsening of severe, malignant osteoporosis (SMO). Regarding current ACTIMMUNE sales, the company stated that “ACTIMMUNE continues to see double digit quarter over quarter net sales growth in the current approved indications, Chronic Granulomatous Disease (CGD) and severe, malignant osteopetrosis (SMO).”

In addition to CGD, SMO and now FA, Horizon has also engaged in other studies to analyze the effectiveness of ACTIMMUNE with different dosages for the treatment of other orphan diseases, including osteoporosis (both intermediate osteoporosis and autosomal dominant osteopetrosis), severe atopic dermatitis and cutaneous T-cell lymphoma.