A breakthrough motor neurone disease drug will become dramatically cheaper for eligible Australians when Qalsody is added to the Pharmaceutical Benefits Scheme on 1 October, cutting the cost of a prescription from about $28,000 to $25.
The treatment is aimed at people with the rare SOD1 genetic mutation linked to amyotrophic lateral sclerosis (ALS), a form of motor neurone disease that causes progressive muscle weakness, paralysis and death.
Qalsody, also known as tofersen, is the first targeted treatment available for this specific genetic form of the condition. Around 70 Australians are expected to benefit from the PBS listing each year.
Motor neurone disease affects almost 3,000 people across Australia and has no cure. The announcement comes after a series of high-profile diagnoses and losses within the country’s sporting community, including the death of campaigner Neale Daniher in May and former NRL player Jai Arrow’s recent diagnosis.
Luke Edmondson was diagnosed at 28, shortly after the birth of his child. He has been taking Qalsody for two and a half years and said he had seen little change in his symptoms.
“My diagnosis was devastating shock at the time, being a 28-year-old with a new baby,” he said. “I’ve noticed little to no progression of my symptoms.”
The drug is administered through a lumbar puncture, with treatment taking place under the care of trained medical professionals. Australia’s Therapeutic Goods Administration granted Qalsody provisional approval in April for adults with ALS associated with an SOD1 mutation.
The regulator said the decision was based on evidence including effects on clinical function, breathing, muscle strength, survival and markers of nerve damage. Continued approval will depend on further data.
MND Australia and FightMND welcomed the PBS decision, but stressed that Qalsody would not help everyone living with the disease. The organisations also urged people with MND to discuss genetic testing with their neurologist, as identifying an SOD1 mutation may determine whether the treatment is suitable.
The groups described the listing as important progress while noting that families affected by other forms of motor neurone disease are still waiting for new treatments, improved care options and, ultimately, a cure.
