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Duchenne Muscular Dystrophy: Orphan Drug Designation In Australia

December 15, 2013

This is the second Blog Post in a series over the next week that will examine Duchenne Muscular Dystrophy (DMD) in the rare disease and orphan drug space.

Australia’s Therapeutic Goods Administration (TGA)  announces on December 10, 2013, the orphan drug designation for Prosensa/GlaxoSmithKline’s Drisapersen, for the treatment of patients with DMD, “bearing certain mutations that are amenable to treatment with exon 51 skipping.”

In Australia, the administrative authority involved in orphan drug regulations, is the Therapeutic Goods Administration (TGA). TGA is part of the Australian government’s Department of Health & Ageing. The following are a few features of orphan drugs in Australia (per the OrphaNet portal) :

•   Orphan Drug Policy set up in 1998

•   Orphan designation is intended for drugs for rare diseases with prevalence of < 2,000 patients in the total population of 18 million OR approximately 1 in 10,000

•   Waiver of application and evaluation and no annual registration fees

•   5-year exclusivity (similar to other drugs)

•   All costs of the orphan drug designation process is covered by TGA

•   R&D not supported by grants or tax incentives.

The alphabetical list of designated orphan drugs in Australia from the TGA is updated as of December 13, 2013.

Please Note: “Technician Transfer Liquid To Test Tube” courtesy of US National Institute of Mental Health.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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