SOURCE: Horizon Pharma plc
Phase 2 Clinical Study in Friedreich's Ataxia Patients Shows Strong Efficacy Signal; Management Estimates Peak Net Sales Potential for ACTIMMUNE of $500 Million With FA Approval
KING OF PRUSSIA, PA--(Marketwired - Oct 13, 2014) - Horizon Pharma plc (NASDAQ: HZNP), a specialty biopharmaceutical company with a portfolio of products in arthritis, inflammation and orphan diseases, is hosting analysts and investors this evening to discuss the opportunities for ACTIMMUNE (interferon gamma-1b).ACTIMMUNE is currently approved by the U.S. Food and Drug Administration (FDA) to treat two ultra-rare orphan diseases, Chronic Granulomatous Disease (CDG) and severe, malignant osteopetrosis (SMO).
The meeting is being conducted in King of Prussia, PA to coincide with the Children's Hospital of Philadelphia (CHOP) 7th Annual Friedreich's Ataxia (FA) Symposium.The results from the Phase 2 clinical trial, sponsored by Friedreich's Ataxia Research Alliance (FARA) and conducted by the Collaborative Clinical Research Network in Friedreich's Ataxia (CCRN in FA), were presented at the CHOP event and are being discussed at the Horizon analyst day.
Highlights from the Horizon Pharma meeting include:
"We are committed to helping patients and physicians identify the early signs and symptoms of CGD and SMO to ensure patients receive more effective treatment sooner," said Timothy P. Walbert, chairman, president and chief executive officer."We also believe there is a significant potential opportunity in Friedreich's ataxia patients and we are committed to working closely with FARA and CCRN in FA to expeditiously initiate the ACTIMMUNE Phase 3 clinical trial."
About Friedreich's Ataxia FA is a debilitating, life-shortening and degenerative neuro-muscular disorder that affects about one in 50,000 people in the United States.Onset of symptoms can vary from five years old to adulthood, with the childhood onset tending to be associated with a more rapid progression.A progressive loss of coordination and muscle strength leads to motor incapacitation and often the full-time use of a wheelchair. Most young people diagnosed with FA require mobility aids such as a cane, walker or wheelchair by their teens or early 20's.There are currently no approved treatments for FA.For more information about FA, visit the Friedreich's Ataxia Research Alliance (FARA) website at http://www.curefa.org.
About ACTIMMUNE (interferon gamma-1b) ACTIMMUNE 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 ofa 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).
Important Safety Information ACTIMMUNE is contraindicated in patients who develop or have known hypersensitivity to interferon-gamma, E. coli-derived products or any component of the product.
The most common adverse experiences occurring with ACTIMMUNE therapy are "flu like" or constitutional symptoms such as fever, headache, chills, myalgia, or fatigue, which may decrease in severity as treatment continues.Some of the "flu-like" symptoms may be minimized by bedtime administration of ACTIMMUNE. Acetaminophen may be used to prevent or partially alleviate the fever and headache.
Link:
Horizon Pharma plc Reviews Future ACTIMMUNE(R) Opportunities at Analyst Day