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 14 August 2014 for the treatment of cystic fibrosis (CF) in patients who have a CFTR mRNA Splice Site (Class V) mutation or a residual function CFTR mutation in the CFTR gene.
The dose form of Ivacaftor (KALYDECO) for this indication is tablets.
The sponsor of Ivacaftor (KALYDECO) is Vertex Pharmaceuticals Australia Pty Ltd.
(Signed by)
Dr Anthony Gill
Delegate of the Secretary
14 August 2014