Onpattro available at lower cost for FAP eligible patients in Australia
Therapy added to public program allowing for subsidized medicines
Onpattro (patisiran), an approved treatment for familial amyloid polyneuropathy (FAP), is now listed on Australia’s Pharmaceutical Benefits Scheme (PBS), meaning that eligible patients in the country can access the therapy at a lower cost.
The listing specifically covers Onpattro’s use by adults with a genetic FAP diagnosis who have stage 1 or stage 2 polyneuropathy, indicating mild to moderate symptoms of damage to multiple nerves.
That’s according to an announcement from Medison Pharma, which is commercializing Onpattro in Australia as part of a partnership with the therapy’s developer, Alnylam Pharmaceuticals.
“Medison welcomes the listing of ONPATTRO on the PBS for Australians who are battling with this devastating disease,” Adam Maguire, country manager of Medison Pharma Australia, said in a company press release.
Prices of medicines listed on Australia’s scheme are lower for eligible patients
The decision follows a recommendation from the country’s Pharmaceutical Benefits Advisory Committee to list Onpattro on PBS as a treatment for FAP, assuring a subsidized price, after further negotiations between Alnylam Australia and the Australian government.
The therapy was approved for use in Australia in November 2022, but the government now will cover much of Onpattro’s cost, with patients generally reported to pay AU$31.60 per prescription (available as 5 mL vials). By comparison, when Onpattro was approved in the U.S. in 2018, it was estimated to cost patients there $345,000 annually.
FAP is a type of hereditary transthyretin amyloidosis (abbreviated as hATTR or ATTRv), which is a broader term referring to diseases caused by mutations in the TTR gene, which lead to the production of an abnormal form of the transthyretin protein.
In FAP, the abnormal protein builds in the body’s tissues, mainly in the nerves outside the brain and spinal cord, causing multiple nerve damage, or polyneuropathy, that results in disease symptoms like muscle weakness and abnormal sensations.
Given that the condition is often misdiagnosed, it’s not known exactly how many Australians have FAP. But according to the Australian Amyloidosis Network (AAN), more than 160 people with a known hATTR diagnosis were seen at clinics between 2007 and 2022, and 40% of them had FAP.
Peter Mollee, MD, chairman and a founding member of the AAN, said the organization is “grateful that the Australian Government has made [Onpattro] available for our patients and their families.”
Better outcomes seen with an early start of disease-modifying treatment
Antonia Carroll, MD, a neurologist and neurophysiologist at St Vincent’s and Westmead Amyloidosis Centres in Australia, noted the “significant unmet need for targeted treatment options in hereditary transthyretin amyloidosis,” adding “that early commencement of disease modifying treatment improves outcomes for these individuals. We are grateful that there is an additional treatment option available for polyneuropathy in this in-need population.”
Onpattro is designed to reduce levels of the abnormal transthyretin protein through a process called RNA interference, or RNAi. It specifically works by targeting the TTR gene’s messenger RNA, an intermediate molecule derived from DNA that is used as a template to manufacture protein.
As such, Onpattro is expected to suppress the production of both the healthy and mutant forms of the protein and prevent the formation of toxic transthyretin clumps, easing patients’ symptoms and slowing FAP progression.
Clinical trials have illustrated that Onpattro, given by infusion into the bloodstream every three weeks, can reduce the severity of nerve damage in FAP and improve patients’ quality of life. Real-world data also has supported the therapy’s ability to prevent further disease progression.
“Following the PBS listing, Australian patients can now access Onpattro, demonstrating Alnylam’s continued commitment to delivering our RNAi therapeutics to patients worldwide,” said Norton Oliveira, senior vice president and head of partner and emerging markets at Alnylam.
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