Therapeutic-area Dossier
Rare Disease
Therapeutic-area Dossier for Rare Disease (rare disease medicines). Australian brands, active ingredients, PBS-listed treatments, PBAC outcomes —…
- Brand: ALYFTREK
- Brand: AMVUTTRA,
- Brand: ANDEMBRY
- Brand: BYLVAY
- Brand: CRYSVITA
- Brand: CYSTADANE
- Sponsor: Sanofi-Aventis
- Sponsor: Takeda Pharmaceuticals
- Sponsor: BioMarin Pharmaceutical
- Sponsor: Vertex Pharmaceuticals
What is
Rare Disease Rare Disease covers therapeutic interventions for uncommon conditions, with 39 brands registered in Australia.
Brands registered in Rare Disease The top brands in this area are:
— CYSTADANE — GALAFOLD — OXLUMO — PHEBURANE — SOHONOS — SYLVANT — ZOLGENSMA — EKTERLY — ALYFTREK — CERDELGA — ORFADIN — TRIENTINE
Sponsor landscape
The rare disease market includes 24 active sponsors. Leading sponsors in this area are Sanofi-Aventis, Dr Reddy's Laboratories, BioMarin Pharmaceutical, Recordati Rare Diseases, Takeda Pharmaceuticals, Vertex Pharmaceuticals, JACE Pharma and Cipla.
PBS and regulatory activity
Seven brands in this area have PBS-listed items, with the latest schedule update dated 1 May 2026. Twenty brands have recorded PBAC outcomes, with the most recent dated 1 July 2025. Fourteen brands have AusPARs on record, with the latest AusPAR dated 14 November 2025.