New life saving medicine free for rare disease

Australians with the extremely rare and life-threatening inherited disease, acid sphingomyelinase deficiency (ASMD) will now have free access to a life saving medicine.
 
Olipudase alfa (Xenpozyme®), an enzyme replacement therapy, will be listed on the Life Saving Drugs Program (LSDP) as the first and only effective treatment approved for Australians with ASMD in children and adult patients with type A/B (Niemann-Pick type A/B) or type B (Niemann-Pick type B).
 
With this genetic mutation, people with ASMD type A/B and B lack a functioning enzyme called acid sphingomyelinase. This essential enzyme is found in parts of the body's cells which break down nutrients and other materials including certain fats.
 
If these fats build up, it changes the way cells work and causes them to die, affecting normal functioning of tissues and organs, including the liver, spleen, lungs, heart and brain.
 
The LSDP is separate to the Pharmaceutical Benefits Scheme (PBS), providing free access to highly specialised medicines to treat patients with ultra-rare and life-threatening diseases. 
 
Quotes attributable to Minister Butler:
 
“This treatment will save lives, and bring great financial relief to families affected by ASMD who would be costed out if not for the Life Saving Drugs Program.
 
“The Albanese Government continues to work with pharmaceutical companies to list life saving medicines to help Australians with rare diseases.”