Date: 2014-10-09
Type of information: Presentation of results at a congress
phase: 2
Announcement: presentation of results the 139th Annual Meeting of the American Neurological Association in Baltimore, MD on Monday, October 13 .
Company: Horizon Pharma (Ireland)
Product: Actimmune® (interferon gamma-1b)
Action
mechanism: protein. Actimmune® (interferon gamma-1b) is currently approved for two rare diseases in the United States. It is approved by the FDA to reduce the frequency and severity of serious infections associated with Chronic Granulomatous Disease (CGD), a genetic disorder that affects the functioning of a type of white blood cell of the immune system, neutrophils or phagocytes, leading to recurrent severe bacterial and fungal infections and chronic inflammatory conditions. In addition,Actimmune® is approved by the FDA to slow the worsening of severe, malignant osteopetrosis (SMO), another genetic disorder that affects normal bone formation causing the abnormal accumulation of bone material which tends to narrow the space inside bones where bone marrow is formed. This can cause failure of the bone marrow, leading to a decrease in various blood cells such as red blood cells (anemia) and white blood cells (decreased ability to fight infection).
Disease: Friedreich\'s Ataxia
Therapeutic area: Rare diseases - Neurodegenerative diseases
Country:
Trial
details: This single center, Phase 2 open-label study of 12 children ages five to 17 years with genetically confirmed FA with treatment of up to 12 weeks, evaluated the safety, tolerability and efficacy of Actimmune®. The study measured frataxin levels, a biomarker of disease and clinical measures (Friedreich\'s Ataxia Rating Scale [FARS] score and other neurological evaluations) as efficacy markers. (NCT01965327)
Latest
news: * On October 9, 2014, Horizon Pharma, a specialty biopharmaceutical company with a portfolio of products in arthritis, inflammation and orphan diseases, announced the presentation of data from a Phase 2 clinical study of Actimmune® (interferon gamma-1b) treatment in children with Friedreich\'s ataxia (FA). An abstract of the data has been published in a supplement to the Annals of Neurology and is being presented as a poster during the 139th Annual Meeting of the American Neurological Association in Baltimore, MD on Monday, October 13 . This single center, Phase 2 open-label study of 12 children ages five to 17 years with genetically confirmed FA with treatment of up to 12 weeks, evaluated the safety, tolerability and efficacy of Actimmune®. The study measured frataxin levels, a biomarker of disease and clinical measures (Friedreich\'s Ataxia Rating Scale [FARS] score and other neurological evaluations) as efficacy markers. The results showed Actimmune® was well tolerated with no serious adverse events and two subjects reporting severe events and subsequent dose reductions. The safety findings generally reflected the labeled safety profile for Actimmune®. Changes in frataxin protein levels, the primary study endpoint, were statistically significant in red blood cells, white blood cells and platelets. The magnitude of change observed was small and varied between tissues. Mean improvement in the FARS score, a clinically validated measurement of patient performance and secondary endpoint, was statistically significant (p=0.008) and was equivalent to two years of extension of disease progression. No other changes were observed. \"The highly significant improvement in the FARS score, a key measure of improvement in disease progression, supports further study of ACTIMMUNE in Friedreich\'s ataxia,\" said Timothy P. Walbert , chairman, president and chief executive officer, Horizon Pharma plc. \"We plan to work with the Collaborative Clinical Research Network in Friedreich\'s Ataxia (CCRN in FA) and the Friedreich\'s Ataxia Research Alliance (FARA) to rapidly move into a registration program for ACTIMMUNE in FA. We plan to meet with the FDA this quarter regarding the clinical development and regulatory pathway for ACTIMMUNE in FA.\"
The study was sponsored by FARA and conducted by David Lynch , M.D., Ph.D., professor of neurology at Children\'s Hospital of Philadelphia (CHOP) and principal investigator of the CCRN in FA. Investigational product was provided by Vidara Therapeutics Research Limited , now part of Horizon Pharma plc.
