Friedreich’s Ataxia: Horizon Pharma’s Orphan Drug Gets FDA Fast-Track Designation
Horizon Pharma, a global specialty biopharmaceutical company, announces April 10th that their product Actimmune (Interferon Gamma-1b) is granted FDA’s Fast Track designation for the rare, genetic disease, Friedreich’s Ataxia (FA).
Horizon Pharma submits FDA’s Investigational New Drug (IND) application in February 2015. The company plans on starting a Phase III study in the second quarter of 2015, in collaboration with the Friedreich’s Ataxia Research Alliance (FARA). In October 2014, Actimmune receives FDA Orphan Drug Designation (ODD) for FA:
FDA ODD Database Record For Horizon Pharma’s FA Indication
|Generic Name:||Interferon gamma-1b|
|Orphan Designation:||Treatment of Friedriech’s Ataxia|
|Orphan Designation Status:||Designated|
|FDA Orphan Approval Status:||Not FDA Approved for Orphan Indication|
|Sponsor:||Vidara Therapeutics Research Ltd. Adelaide Chambers Dublin 8 IRELAND **|
** Horizon Pharma completed acquisition of Vidara Therapeutics in September 2014.
FA is a degenerative neuro-muscular disorder that affects approximately 1/50,000 people in the United States. FA can start anywhere from childhood to adulthood. There is loss of muscle strength and coordination. Those patients diagnosed with FA at a young age require mobility aids, such as a wheelchair, walker, or cane by their teens or early 20’s. There are currently no approved treatments for FA.
Actimmune is currently approved in the United States for 2 genetic diseases:
• Chronic Granulomatous Disease (GD) – affects a type of white blood cell of the immune system
• Delaying time to disease progression for Severe , Malignant Osteopetrosis (SMO) – affects normal bone formation.
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