TGA grants orphan drug status to Raxone for DMD

Release Date: 16-Oct-2016

Santhera Pharmaceuticals ‘s lead drug candidate, Raxone, has been approved by Therapeutic Goods Administration (TGA) as an orphan drug to treat patients with Duchenne Muscular Dystrophy (DMD). Raxone, under the name of Catena, has already received same status by the European, Swiss, and U.S regulatory authorities.


Raxone has been designed to show effective results in patients with disfunctioning of respiratory organ. The company describes it as a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H: quinone oxidoreductase (NQO1). The drug is made to stimulate mitochondrial electron transport, reducing and scavenging reactive oxygen species (ROS or free radicals) and supplementing cellular energy that which are highly dependent on mitochondrial activity.


TGA provides investigational treatments for rare disorders that affect less than 2000 people in that country. Also, the drug qualifies for market exclusivity post approval of the designation.


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