COMMONWEALTH OF AUSTRALIA
Department of Health
Therapeutic Goods Administration
THERAPEUTIC GOODS ACT 1989
DESIGNATION OF Lumacaftor (VX-809) 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 Lumacaftor (VX-809) as an orphan drug on 22 September 2014 in combination with Ivacaftor (VX-770) for the treatment of cystic fibrosis (CF) in patients who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
The dose form of Lumacaftor (VX-809) for this indication is tablets.
The sponsor of Lumacaftor (VX-809) is Vertex Pharmaceuticals (Australia) Pty Ltd.
(Signed by)
Dr Anthony Gill
Delegate of the Secretary
22 September 2014