Australia's TGA designates three orphan drugs
This article was originally published in Scrip
Executive Summary
Australia's Therapeutics Goods Administration (TGA) has designated three new orphan drugs. These are: Actelion's Zavesca (miglustat) capsules, for the treatment of Niemann-Pick disease type C, designated on July 8th; Ipsen's Increlex (mecasermin injection 10mg/ml in 40mg vial), for the long-term treatment of growth failure in children and adolescents with severe primary insulin-like growth factor-1 deficiency (severe primary IGFD), designated on July 1st; and Celgene's Vidaza (azacytidine) in a powder for suspension for injection, for the treatment of myelodysplastic syndrome, designated on June 17th.