COMMONWEALTH OF AUSTRALIA
Department of Health
Therapeutic Goods Administration
THERAPEUTIC GOODS ACT 1989
DESIGNATION OF nitisinone (disanit) AS AN ORPHAN DRUG
I, Dr Tim Greenaway, 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 nitisinone (DISANIT) as an orphan drug on 20 July 2017 for the treatment of adult and paediatric (in any age range) patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.
The dose form of nitisinone (DISANIT) for this indication is Capsules 2mg 5mg and 10mg (stable capsule formulation).
The sponsor of nitisinone (DISANIT) is Emerge Health Pty Ltd.
(Signed by)
Dr Tim Greenaway
Delegate of the Secretary
20 July 2017