Two breakthrough medicines will be available free of charge to Australians with extremely rare and life-threatening inherited diseases, from 1 April 2023.
The Australian Government has added the two medicines, Kanuma® (sebelipase alfa) and Cerdelga® (eliglustat) to the Life Saving Drugs Program (LSDP). Both medicines treat enzyme deficiency diseases.
Kanuma is the first effective treatment discovered for infantile onset lysosomal acid lipase deficiency, or LAL-D. Without the treatment, babies born with this condition would not survive more than 12 months.
Cerdelga is listed for treatment of Gaucher disease (Type 1), as an oral alternative to treatments delivered by infusions.
Gaucher disease (Type 1) is caused by deficiency of an enzyme required to break down fats, which build up in the spleen, liver and bone marrow.
Cerdelga will be available for patients aged 18 years and over who have had 12 months prior treatment with one of the 3 other enzyme replacement therapies for Gaucher disease (Type 1) listed on the LSDP, all of which require infusion.
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.
These listings will expand the LSPD to a total of 18 medicines for 11 diseases.
Quotes attributable to Minister Butler:
“Listing these medicines on the Life Saving Drugs Program will save lives, save heartache and save patients hundreds of thousands of dollars a year.
“Australia is one of the first countries in the world to subsidise Kanuma. Parents will no longer have to watch their baby born with LAL-D grow sicker, or literally spend a fortune to buy this drug.
“The listing of Cerdelga will also provide great relief for adults with Gaucher disease type 1, who will be able to take a capsule and get on with their lives instead of going to hospital for an infusion.”