COMMONWEALTH OF AUSTRALIA
Department of Health
Therapeutic Goods Administration
THERAPEUTIC GOODS ACT 1989
DESIGNATION OF Carglumic acid (Udecane) 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 Carglumic acid (Udecane) as an orphan drug on 11 October 2016 for the treatment of:
- Hyperammonaemia due to N-acetylglutamate synthase primary deficiency;
- Hyperammonaemia due to organic acidaemias such as:
- hyperammonaeia due to isovaleric acidaemia
- hyperammonaemia due to methylmalonic acidaemia
- hyperammonaemia due to propionic acidaemia
The dose form of Carglumic acid (Udecane) for this indication is dispersible tablets.
The sponsor of Carglumic acid (Udecane) is Orpharma Pty Ltd.
(signed by)
Dr Anthony Gill
Delegate of the Secretary
11 October 2016