Sydney, Thursday 1 August, 2024. Medison Pharma, alongside their partner Alnylam Pharmaceuticals, Inc., are pleased to announce that from 1 August 2024, ONPATTRO® (patisiran) is listed on the Pharmaceutical Benefits Scheme (PBS) for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients, with stage 1 or stage 2 polyneuropathy, confirmed by genetic testing. [1],[2],[3]
Hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis also referred to as ATTRv) is a rare, rapidly progressing and fatal type of amyloidosis that runs in families and occurs when certain genetic mutations are inherited.3,4,8,9 The disease results in abnormal protein deposits, made of transthyretin (TTR), which accumulate and damage various organs and tissues within the body. This may cause damage to the peripheral nervous system, which is referred to as polyneuropathy. Some patients present with debilitating polyneuropathy that is associated with a rapid progression and worsening quality of life.6,7
Professor Peter Mollee, Chairman and founding member of the Australia Amyloidosis Network (AAN) said, “Amyloidosis is a complex and devastating disease often leading to organ failure. Early and accurate diagnosis is crucial for clinical management, which is best provided via a multidisciplinary team. Hereditary transthyretin-mediated amyloidosis is a rare form of amyloidosis which particularly affects the heart and nerves. It cannot be diagnosed without genetic testing. We are grateful that the Australian Government has made this new treatment option available for our patients and their families.”
While the true prevalence in Australia is unknown, there were 161 people with a known diagnosis of hereditary transthyretin-mediated amyloidosis, who attended the Australian Amyloidosis Network (AAN) clinics between 2007 to 2022, of whom forty percent presented with neuropathic disease alone and 25% with mixed cardiac and neuropathic disease.5,4
Dr. Antonia Carroll, Neurologist and Neurophysiologist, St Vincent’s and Westmead Amyloidosis Centres said, “Hereditary transthyretin amyloidosis (ATTRv) is a devastating, inherited, life-threatening, multiorgan disease which results in poor functional capacity and quality of life for patients and their families. ATTRv remains underdiagnosed in Australia. There is often a significant delay in diagnosis of up to 4 years with patients frequently seeing up to five physicians prior to the diagnosis being made.”
“There is a significant unmet need for targeted treatment options in hereditary transthyretin amyloidosis. We know 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® contains the active ingredient patisiran, which is a small interfering RNA (siRNA), also known as RNA interference (RNAi), which is designed to silence the gene responsible for the expression of TTR. 2,7 ONPATTRO® lowers the amount of TTR protein made by the liver, resulting in less TTR protein in the blood that can form amyloid, and therefore helps to reduce the effects of this illness.3
Norton Oliveira, Senior Vice President and Head of Partner and Emerging Markets at Alnylam, said, “Following the PBS listing, Australian patients can now access ONPATTRO®, demonstrating Alnylam’s continued commitment to delivering our RNAi therapeutics to patients worldwide.”
“We are committed to realizing our vision, ensuring that every patient in need, regardless of where they live, will have fast access to highly innovative therapies. This PBS listing allows Australian patients access to a new treatment for hATTR." said Gil Gurfinkel, CEO of Medison Pharma.
Adam Maguire, Country Manager of Medison Pharma Australia said,
“Medison welcomes the listing of ONPATTRO® on the PBS for Australians who are battling with this devastating disease. We remain committed to ensuring that Australians have access to highly innovative therapies as fast as possible.”
ONPATTRO® (patisiran) 10 mg/5 mL concentrated injection for infusion vial 3
Indication: ONPATTRO® is a prescription medicine used for the treatment of polyneuropathy caused by an illness called hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults. It is used only in adults with stage 1 or stage 2 polyneuropathy.
Dosage and Method of Administration:
ONPATTRO® treatment is initiated via an intravenous (IV) infusion under the supervision of a physician knowledgeable in managing amyloidosis once every 3 weeks. The dose is based on a patient’s actual body weight.
Precautions & Interactions:
A doctor will determine if ONPATTRO® is a suitable treatment option for a patient with hATTR amyloidosis. The doctor will consider the patient’s other medical conditions and medicines, including pregnancy and breastfeeding. All patients should receive premedication before initiating ONPATTRO® administration to reduce the risk of infusion-related reactions (IRRs). Vitamin A supplementation is advised for patients treated with ONPATTRO®.
Adverse Effects: All medicines can have side effects. Most common side effects of ONPATTRO® include swelling of the arms and legs, irritation or infection of the airways (such as colds, sinus infections, and nasal congestion) and infusion-related reactions, symptoms of which include body aches or pain, reddening of the face or body, nausea, stomach pain, feeling short of breath or cough, hoarseness of voice, chest discomfort or chest pain, headache, warm skin, rash, itching, chills, dizziness, feeling tired, rapid heart rate, low or high blood pressure which may include fainting, swelling of the face, and pain, redness, burning sensation, or swelling at or near the infusion site.
For further information, patients should speak to their doctor and can refer to the ONPATTRO® (patisiran) Consumer Medicine Information available at https://www.medisonpharma.com.au/products/OnpattroCMI or https://www.ebs.tga.gov.au/ebs/picmi/picmirepository.nsf/pdf?OpenAgent=&id=CP-2024-CMI-02187-1
PBS Information: Section 100 Highly Specialised Drug (HSD) Program for adult patients with stage 1 or stage 2 polyneuropathy caused by hereditary transthyretin amyloidosis.
Authority Required. Please refer to PBS Schedule for full authority information.
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This medicinal product is subject to additional monitoring in Australia. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse events at www.tga.gov.au/reporting-problems.
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ENDS
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Media Inquiries: Adam Maguire | M: 0431001020 | E: Adamm@medisonpharma.com
Disclosure Medison supports disclosure and transparency in interactions between personnel and healthcare professionals to ensure public trust and confidence.
No spokespeople have been offered compensation for their involvement in this media release. All spokespeople have been briefed on the approved use of this product and their obligations regarding promotion to the general public.
ONPATTRO® is a registered trademark of Alnylam Pharmaceuticals, Inc. © 2024 Medison Pharma Australia Pty Ltd, ABN 19 659 723 403, 1-7 Bligh Street, Sydney NSW 2000, www.medisonpharma.com.au
Prepared July 2024, [AUS-ALNY-P-240007].
About Medison Pharma
Medison, the creator and leader of the global partnership category, is a global pharma company focused on providing access to highly innovative therapies to patients in international markets.
Medison is the first to create an international commercialization platform for highly innovative therapies, helping to save and improve lives by making the best available novel treatments accessible to patients in international markets. Medison has a track record of global partnerships with leading pharmaceutical and biotech companies seeking to expand their global reach.
To learn more visit www.medisonpharma.com.
About Alnylam
Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. RNAi therapeutics represent a clinically validated approach that has yielded five Alnylam-discovered medicines that have received marketing authorization in markets around the world. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is headquartered in Cambridge, MA. For more information about Alnylam please visit www.alnylam.com
About Medison’s Partnership with Alnylam
Medison has a global partnership with Alnylam to commercialise RNAi Therapeutics in various markets such as Israel, Europe, Latin America, and Asia-Pacific, including Australia. This partnership reflects Medison’s and Alnylam’s belief that every patient, wherever they are in the world, deserves a fair chance to receive disease modifying, highly innovative therapies.
References:
[1]. Australian Government Department of Health and Aged Care. Schedule of Pharmaceutical Benefits Summary of Changes Effective 1 August 2024. www.pbs.gov.au.
[2]. Onpattro Product Information, July 2024. Available at: www.ebs.tga.gov.au www.ebs.tga.gov.au
[3]. Onpattro Consumer Medicine Information, July 2024. Available at: www.ebs.tga.gov.au www.ebs.tga.gov.au
- Australian Amyloidosis Network. Hereditary amyloidosis. Available at: https://aan.org.au/patients-and-carers/hereditary-amyloidosis/ (Accessed July 11, 2024).
- Carroll AS, Kwok F, Bennetts B, et al. 2749 The spectrum of neuropathy in hereditary transthyretin amyloidosis (ATTRv) in Australia. BMJ Neurology Open2023;5 [Poster Abstract] doi: 10.1136/bmjno-2023-ANZAN.128.
- Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis 8, 31 (2013). https://doi.org/10.1186/1750-1172-8-31
- Adams D, Gonzalez-Duarte A, O’Riordan WD, et al. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N Engl J Med 2018;379:11-21 doi: 10.1056/NEJMoa1716153
- Philip N. Hawkins et al. Evolving landscape in the management of transthyretin amyloidosis. Annals of Medicine 2025;doi: 10.3109/07853890.2015.10689499.
- Buxbaum, Joel N., Angela Dispenzieri, David S. Eisenberg, Marcus Fändrich, Giampaolo Merlini, Maria J. M. Saraiva, Yoshiki Sekijima, and Per Westermark. Amyloid Nomenclature 2022: Update, Novel Proteins, and Recommendations by the International Society of Amyloidosis (ISA) Nomenclature Committee. Amyloid 29 (4): 213–19. doi:10.1080/13506129.2022.2147636.
- Lane, Thirusha et al. “Natural History, Quality of Life, and Outcome in Cardiac Transthyretin Amyloidosis.” Circulationvol. 140,1 (2019): 16-26. doi:10.1161/CIRCULATIONAHA.118.038169