A life-saving drug for a rare hereditary disease is now free for Australians.
Federal Health Minister Mark Butler said Olipudase alfa (Xenpozyme®) would be listed on the Life Saving Drugs Program (LSDP) as the first and only effective treatment approved for Australians with the life-threatening inherited disease, acid sphingomyelinase deficiency (ASMD).
Minister Butler said the drug, an enzyme replacement therapy, was approved for children with ASMD and adult patients with type A/B (Niemann-Pick type A/B) or type B (Niemann-Pick type B).
He said with this genetic mutation, people with ASMD type A/B and B lacked 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.”
Minister Butler said the LSDP was separate to the Pharmaceutical Benefits Scheme (PBS), providing free access to highly specialised medicines to treat patients with ultra-rare and life-threatening diseases.
“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.”
