Life-saving drug for adult Pompe patients to be funded in Australia

Australia’s Minister for Health Sussan Ley has approved a life-saving drug that will ensure adult Australian Pompe patients receive treatment that previously would have cost several hundred thousand dollars at no out-of-pocket cost.

Ms Ley said the government would commit close to A$40 million (around $29 million) over five years to list the drug Myozyme (alglucosidase alfa) from Sanofi (Euronext: SAN) subsidiary Genzyme, for patients over 18 years with late-onset Pompe disease through the Life Saving Drugs Programme (LSDP) from 1 September 2015.

Already listed for younger patients

Earlier this year Myozyme was approved for subsidization for children aged two to 18 years under the Life Saving Drugs Program (The Pharma Letter February 17).

“Pompe disease is a rare and severe medical condition, affecting only a very small number of Australians every year,” Ms Ley said, adding: “However, it is a very expensive treatment – costing several hundred thousand dollars – putting it out of reach for many Australians. This decision will improve the lives of adult patients with late-onset Pompe disease.”

This approval is the result of considerable effort on behalf of the Australian Pompe Association, the sponsoring company Genzyme and officers of the Health Department to bring together the necessary evidence to support this listing. The treatment is available now with the government subsidy to begin from September 1 following Ms Ley’s approval of the new indication of use for the medicine.