Skip to content

Friedreich’s Ataxia: Horizon Pharma’s Orphan Drug Gets FDA Fast-Track Designation

April 13, 2015

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
Trade Name: Actimmune
Date Designated: 10-01-2014
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.


Friedreich’s Ataxia Research Alliance (FARA)

National Ataxia Foundation.

Please Note: “LuMaxArt Golden Guys Teamwork Gear Runners” by lumaxart [CC-BY-SA-2.0] | Wikimedia Commons.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

Leave a Comment

Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out /  Change )

Google photo

You are commenting using your Google account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )

Connecting to %s

%d bloggers like this: