COMMONWEALTH OF AUSTRALIA
Department of Health
Therapeutic Goods Administration
THERAPEUTIC GOODS ACT 1989
DESIGNATION OF Drisapersen AS AN ORPHAN DRUG
I, Dr Anthony Gill, Delegate of the Secretary for the purposes of 16J of the Therapeutic Goods Regulations 1990 (“the Regulations”), acting under subregulation 16J(2) of the Regulations, designate Drisapersen as an orphan drug on 10 December 2013 for the treatment of patients with Duchenne muscular dystrophy (DMD) bearing certain mutations that are amenable to treatment with exon 51 skipping.
The dose form of Drisapersen for this indication is solution for injection
The sponsor of Drisapersen is GlaxoSmithKline.
(Signed by)
Dr Anthony Gill
Delegate of the Secretary
10 December 2013