Horizon Pharma PLC starts Phase 3 Trial of ACTIMMUNE for treating People With Friedreich's Ataxia Print E-mail
By Marilyn Mullen   
Monday, 08 June 2015 14:14

Biopharmaceutical firm Horizon Pharma PLC. (NASDAQ:HZNP) has started Phase 3 Safety, Tolerability and Efficacy of ACTIMMUNE Dose Escalation in Friedreich's Ataxia study for treating people with a degenerative neuro-muscular disorder, Friedreich's Ataxia.


Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma, opined that since it is a rare disease and hence Friedreich's Ataxia shows an area of high medical need.


He added that Phase 3 study shows huge improvement in assessing ACTIMMUNE in the population that is about four thousand people in United States. 


Walbert added that he is looking forward on continuing work with FDA on achieving potential treatment option to FA community.


The Phase 3 trial is a randomized and placebo-controlled study with patients randomized 1:1 for getting subcutaneous doses of placebo three times a week for about 26 weeks. 


There would be enrolment of 90 patients at four sites in United States whereas the first endpoint would gauge change in neurological outcome besides assessing effect of ACTIMMUNE versus placebo which is focused on objective neurologic measures like upper and lower extremity coordination improvement from baseline. 


Additionally, STEADFAST trial would assess pharmacokinetic characteristics of ACTIMMUNE in population with FA.


Ronald J. Bartek, president, director and co-founder, Friedreich's Ataxia Research Alliance, opined that the clinical trial is example of progress made by scientific community in understanding FA.


He added that until two decades back, there was no cause identified for FA whereas now there is late-stage clinical study bringing the firm closer to first treatment for people with FA.


ACTIMMUNE was granted Fast Track status in April 2015 for FA by U.S. Food and Drug Administration.


The designation allows more communication with FDA throughout drug development as well as review process with the aim of increasing approval. 


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