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Australia Insists Orphan Designations Will Last Only Six Months, Overrides Industry Objections

 
• By Neena Brizmohun

Sponsors making drugs for rare diseases in Australia who fail to file for approval within six months of being granted orphan designation will see their designation disappear, the Therapeutic Goods Administration has decided. The new procedure will be problematic for orphan drug sponsors and might even make it more difficult for them to use the new expedited pathways the agency is planning to introduce for innovative drugs.

Map of Australia in pills
Australian orphan drug designations are to lapse after just six months

Rare disease drug makers will need to get their skates on when it comes to applying for market approval in Australia, after the Therapeutic Goods Administration confirmed it would be setting a six-month expiration date on orphan drug designations.

The TGA is replacing its system of granting orphan designations indefinitely despite concerns from drug sponsors that a six-month deadline is too short and that a deadline of

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