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Current Approved Orphan Drugs


Currently, there are 15 listed treatments available for 10 different rare diseases which are funded via the Life Saving Drugs Program.


In order for a patient to qualify for funded treatment, they must meet a certain criteria and apply with support of their treating doctor. 

 

Imiglucerase (Cerezyme®), Velaglucerase (VPRIV®), Taliglucerase (Elelyso®) and Miglustat (Zavesca®) for the treatment of Gaucher disease (Type 1)

 

Agalsidase alfa (Replagal®), Agalsidase beta (Fabrazyme®) and Migalastat (Galafold®) for the treatment of Fabry Disease


Laronidase (Aldurazyme®) for the treatment of Mucopolysaccharidosis Type I (MPS I)


Idursulfase (Elaprase®) for the treatment of Mucopolysaccharidosis Type II (MPS II)

 


Galsulfase (Naglazyme®) for the treatment of Mucopolysaccharidosis Type VI (MPS VI) 

 

Alglucosidase alfa (Myozyme®) for the treatment of Infantile-onset, Juvenile Late-onset or Adult Late-onset Pompe Disease

 

Eculizumab (Soliris®) for the treatment of Paroxysmal Nocturnal Haemoglobinuria (PNH) 

 

Nitisinone (Orfadin®) for the treatment of Hereditary Tyrosinaemia Type I

 

Brineura® (Cerliponase Alfa®) for the treatment of Batten Disease

 

Source: http://www.health.gov.au/lsdp