COMMONWEALTH OF AUSTRALIA
Department of Health
Therapeutic Goods Administration
THERAPEUTIC GOODS ACT 1989
DESIGNATION OF Ivacaftor (KALYDECO) 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 Ivacaftor (KALYDECO) as an orphan drug on 24 November 2014 for the treatment of cystic fibrosis (CF) in patients aged 2 to 5 years old who have a gating (class III) or R117H, Residual or mRNA Splice Site (Class V) mutation in the CFTR gene.
The dose form of Ivacaftor (KALYDECO) for this indication is granules.
The sponsor of Ivacaftor (KALYDECO) is Vertex Pharmaceuticals (Australia) Pty Ltd.
(Signed by)
Dr Anthony Gill
Delegate of the Secretary
24 November 2014