AMVUTTRA® (vutrisiran) is now listed on the Pharmaceutical Benefits Scheme (PBS) for eligible Australians with hereditary transthyretin-mediated amyloidosis with polyneuropathy [1] - Medison

1 May 2025. Medison Pharma Australia Pty Ltd (“Medison Pharma”), alongside their partner Alnylam Pharmaceuticals, Inc. (“Alnylam”), is pleased to announce that from 1 May 2025, AMVUTTRA® (vutrisiran) is listed on the Pharmaceutical Benefits Scheme (PBS) for hereditary transthyretin-mediated amyloidosis in adult patients, with stage 1 or stage 2 polyneuropathy, confirmed by genetic testing.^1,2,3

Hereditary transthyretin-mediated amyloidosis (ATTRv, sometimes written as hATTR) is a rare form of amyloidosis that is inherited within families. ^3,4,8 This condition arises from specific genetic mutations that lead to production by the liver, of abnormal protein deposits composed of transthyretin (TTR). These deposits, known as amyloid, build up in various organs and tissues and can cause damage or stop them from working normally. Polyneuropathy, when the peripheral nervous system is damaged, is a possible and significant consequence of ATTRv. Some patients present with rapidly progressing, severe polyneuropathy, which leads to a deterioration in their quality of life and is ultimately fatal.^5,6,7

Professor Peter Mollee, Chairman and founding member of the Australia Amyloidosis Network (AAN), said, “Hereditary transthyretin-mediated (ATTRv) amyloidosis is a rare and serious disease affecting the heart and nerves. With the availability of vutrisiran on the PBS, patients with ATTRv amyloidosis with polyneuropathy will have access to treatment, which is given as a subcutaneous injection every three months. We’re grateful the Australian Government has made this therapy available to patients and their families”

Genetic testing is required to diagnose hereditary transthyretin-mediated amyloidosis (ATTRv).⁴ Genetic testing is available for patients in Australia and identifies specific mutations in the transthyretin (TTR) gene to confirm the diagnosis.^1,4Early detection through genetic testing in consultation with the treating physician allows for optimal management and timely treatment of ATTRv.^1,4,6

Dr. Antonia Carroll, Neurologist and Neurophysiologist, St Vincent’s and Westmead Amyloidosis Centres said, “Hereditary Transthyretin Amyloidosis (ATTRv) is a complex, inherited condition with devastating outcomes for patients involving multiple organs, including the peripheral nerves and heart. When involving the nerves, this results in significant sensory disturbance, pain, weakness and functional impairments. Genetic testing is essential to diagnose ATTRv in patients, and family members who may be affected. Patients are often unable to work with poor quality of life and significant care needs. We are grateful that the Australian Government has made another treatment option accessible to our patients with ATTRv with polyneuropathy.”

AMVUTTRA® is a medication used to treat polyneuropathy in adults with hereditary transthyretin-mediated amyloidosis (ATTRv) and contains the active ingredient vutrisiran, a small interfering RNA (siRNA). It uses RNA interference (RNAi) to silence the gene responsible for producing the disease-causing protein, transthyretin (TTR), in the liver.^2,6 This reduces the levels of TTR protein in the blood that can form amyloid, and in doing so, helps reduce the effects of this illness.³

Norton Oliveira, Senior Vice President and Head of Partner and Emerging Markets at Alnylam, said, “We are thrilled that AMVUTTRA® has been approved in Australia, offering patients a new treatment option for this rare disease. We are proud of our global partnership with Medison that brings this therapy to Australia.”

“The reimbursement of AMVUTTRA® in Australia is a testament to our commitment to making therapies available to patients with rare and severe diseases, no matter where they live. This milestone reflects the strength of our global partnership with Alnylam, demonstrating the impact of our global commercialization platform." said Victor Papamoniodis, Chief Commercial Officer at Medison Pharma.

Adam Maguire, Country Manager of Medison Pharma Australia, said, “We are proud to bring AMVUTTRA® to Australian patients, offering another treatment option for those living with ATTRv/hATTR. This reimbursement decision reaffirms our mission through collaborative efforts with the local healthcare ecosystem.”

AMVUTTRA® (vutrisiran) 25 mg vutrisiran in 0.5 mL solution³

Indication: AMVUTTRA® 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: AMVUTTRA® treatment is initiated via an injection under the skin every 3 months under the supervision of a physician knowledgeable in managing amyloidosis. The injection will be given in the stomach area (abdomen), upper arm or thigh.

Contraindication: Do not use AMVUTTRA® if you are allergic to vutrisiran or any of the other inactive ingredients (Monobasic sodium phosphate dihydrate, Dibasic sodium phosphate dihydrate, Sodium chloride, Water for injections, Sodium hydroxide, Phosphoric acid).

Warning and Precautions: A doctor will determine if AMVUTTRA® 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. Vitamin A supplementation is advised for all patients treated with AMVUTTRA® because it lowers the amount of vitamin A in the blood. Signs of low vitamin A may include: sight problems especially at night, dry eyes, hazy, or cloudy vision.

Interactions: Tell your doctor or pharmacist if you are taking any other medicines, including any medicines, vitamins or supplements that you buy without a prescription from your pharmacy, supermarket or health food shop.

Adverse Effects: All medicines can have side effects. Most common side effects of AMVUTTRA® include painful joints and pain in the arms and legs (muscle, bone and soft tissue related), shortness of breath (breathing related) and local reactions which includes redness, pain, itching, bruising, or warmth where the injection was given.

For further information, patients should speak to their doctor and can refer to the AMVUTTRA® (vutrisiran) Consumer Medicine Information available at www.medisonpharma.com.au/products/AmvuttraCMI

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 the PBS Schedule for full authority information.

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.

ENDS

_________________________________________________________________________________________

Media Inquiries: Adam Maguire | M: 0431001020 | E: Adamm@medisonpharma.com

Disclosure: Medison Pharma 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. Medison Pharma gained the permission of all spokespeople to add their quotations before publication.

Prepared April 2025, [AUS-ALNY-NP-250001].

About Medison Pharma

Medison, the creator and leader of the global partnership category, is a global commercial platform 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 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 Pharma’s Partnership with Alnylam

Medison Pharma has a global partnership with Alnylam to commercialise RNAi therapeutics in various markets, including Asia-Pacific (including Australia) Europe, Israel, and Latin America. This partnership reflects Medison Pharma’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:

____________________________________

Australian Government Department of Health and Aged Care. Schedule of Pharmaceutical Benefits Summary of Changes Effective 1 May 2025 . Pharmaceutical Benefits Scheme (PBS) | New,amended and deleted
Amvuttra Product Information, April 2025. Available at: www.medisonpharma.com.au/products/AmvuttraPI
Amvuttra Consumer Medicine Information, April 2025 Available at: www.medisonpharma.com.au/products/AmvuttraCMI
Australian Amyloidosis Network. Hereditary amyloidosis. Available at: Hereditary Amyloidosis - Australian Amyloidosis Network (Accessed April 2025).
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
David Adams, Ivailo L. Tournev, Mark S. Taylor, Teresa Coelho, Violaine Planté-Bordeneuve, John L. Berk, Alejandra González-Duarte, Julian D. Gillmore, Soon-Chai Low, Yoshiki Sekijima, Laura Obici, Chongshu Chen, Prajakta Badri, Seth M. Arum, John Vest, Michael Polydefkis & The HELIOS-A Collaborators (2023) Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial, Amyloid, 30:1, 18-26, DOI: 10.1080/13506129.2022.2091985
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.