1 October 2025. Medison Pharma Australia Pty Ltd (“Medison”), alongside their partner Alnylam Pharmaceuticals, Inc. (“Alnylam”), is pleased to announce that from October 1st 2025, OXLUMO® (lumasiran) is listed on the Pharmaceutical Benefits Scheme (PBS) for primary hyperoxaluria type 1 (PH1). 1,2,3
Primary hyperoxaluria type 1 (PH1) is a rare disease that is inherited within families in which the liver produces too much of a substance called oxalate3,4. The kidneys remove oxalate from the body and it is passed out in the urine3. In people with PH1, the extra oxalate can build up in the kidneys, causing kidney stones and progressive kidney damage4,5. A build-up of oxalate can also damage other parts of the body, such as the eyes, heart, skin and bone, and this is called oxalosis3,5. Although PH1 affects patients of all ages, the onset of symptoms occurs at a young age during childhood or adolescence in the majority of patients6.
Prof. Andrew Mallett, Consultant Nephrologist and National Director of the KidGen Collaboration, said, “Primary hyperoxaluria is a complex, rare and hereditary condition that can affect multiple organ systems, including the kidneys. This can result in progressive decline in kidney function, including potential for kidney failure and recurrence of the condition in a kidney transplant. Kidney disease, including that related to primary hyperoxaluria, has a very substantial impact on the quality and quantity of life for those affected by it. Lumasiran is a treatment for primary hyperoxaluria type 1 and we are thankful that the Australian Government has made this therapy available to Australian patients and families affected by this condition.”
A/Prof. John Whitlam, Director of Transplantation and Lead of Renal Genetics, Austin Health, said, “Primary hyperoxaluria type 1 (also known as PH1) is a rare inherited condition in which excess oxalate production by the liver leads to recurrent kidney stones, progressive kidney damage, and in many cases, kidney failure. When organ failure occurs, liver and kidney transplantation may be needed. Many people with PH1 undergo multiple procedures and face limited treatment options. A treatment that slows the accumulation of oxalate is an important step towards slowing down the damage caused by this condition. We’re grateful the Australian Government has made this therapy available to patients and their families.”
OXLUMO® is a medication used to treat primary hyperoxaluria type 1 in all age groups and contains the active ingredient lumasiran, a small interfering ribonucleic acid (siRNA)2. It targets the body’s oxalate production processes through RNA interference3. Lumasiran reduces the amount of an enzyme called glycolate oxidase that the liver makes, which is one of the enzymes involved in producing oxalate3. This reduces the oxalate levels in urine and plasma, and in doing so, helps to reduce the effects of the illness3.
Norton Oliveira, Senior Vice President and Head of Partner and Emerging Markets at Alnylam, said, “We are delighted that patients in Australia now have access to OXLUMO®, offering a new treatment option for those living with primary hyperoxaluria type 1. Through our strong global collaboration with Medison, we aim to deliver renewed hope to families affected by this rare disease.”
“The PBS listing of OXLUMO in Australia reflects our broader commitment to accelerating access to breakthrough therapies for patients with rare diseases and severe diseases. We are proud to collaborate with Alnylam to bring this treatment to patients in Australia,” said Victor Papamoniodis, Chief Commercial Officer at Medison.
Adam Maguire, Country Manager, Australia at Medison, said. “We are proud to help deliver meaningful impact to the rare disease community in the country, and with this listing, eligible Australian patients affected by primary hyperoxaluria type 1 will now be able to access a targeted therapy.”
Each vial contains OXLUMO® (lumasiran) 0.5 mL of lumasiran sodium solution equivalent to 94.5 mg lumasiran. Each mL solution contains lumasiran sodium equivalent to 189 mg lumasiran2,3
Indication: OXLUMO® is indicated for the treatment of primary hyperoxaluria type 1 (PH1) in all age groups.
Dosage and Method of Administration: OXLUMO® is administered as a subcutaneous (under the skin) injection. The OXLUMO® injection should be given to you by a trained healthcare professional. Your doctor will calculate the dose for you based on your body weight and phase of treatment. Initially, you will be given OXLUMO® once a month, which may reduce in frequency after 3 months of treatment.
Contraindication: Do not use OXLUMO® if you are allergic to lumasiran or any of the other inactive ingredients (Sodium hydroxide, Phosphoric acid, Water for injections).
Warnings and Precautions: A doctor will determine if OXLUMO® is a suitable treatment option for their patient with hyperoxaluria type 1 (PH1). The doctor will consider your other medical conditions and medicines, including pregnancy and breastfeeding. Check with your doctor if you have severe or end-stage kidney impairment or moderate to severe liver disease, which may require monitoring.
Interactions: Tell your doctor or pharmacist if you are taking any other medicines, including any medicines, vitamins or supplements 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 OXLUMO® include stomach pain, discomfort or tenderness, local reaction at the injection site (redness of the skin, swelling, pain, itching, rash, bruising) and a type of allergic reaction (hypersensitivity) with symptoms such as rash, throat irritation, and watery eyes.
For further information, patients should speak to their doctor and can refer to the OXLUMO® (lumasiran) Consumer Medicine Information available at www.medisonpharma.com.au/products/OxlumoCMI
PBS Information: GENERAL – General Schedule (Code GE) for patients with primary hyperoxaluria type 1
Authority Required. Please refer to the PBS Schedule for full authority information.
| This medicine is subject to additional monitoring in Australia. This will allow quick identification of new safety information. You can help by reporting any side effects you may get. You can report side effects to your doctor, or directly at www.tga.gov.au/reporting-problems. |
ENDS
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For more information, please contact:
Medison Communications at corpcomms@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.
OXLUMO® 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 September 2025, [AUS-ALNY-NP-250031].
About Medison Pharma
At Medison, we are addressing one of the pharma industry’s most pressing challenges: enabling access to breakthrough therapies for patients with severe and rare diseases in international markets where traditional commercialization models fall short.
As the creator and leader of the global partnership category, we have built a first-of-its-kind unified commercialization platform that enables biotechs to effectively plan, optimize and launch their therapies on a global scale.
With a strong leadership team and a proven track record, we are the partner of choice for innovative biotech companies seeking to expand beyond the US, Western Europe and Japan into regions where most of the world’s population resides. Our affiliate-like model integrates commercial, medical and operational capabilities across diverse geographies, accelerating access and delivering life-changing treatments to more patients, faster.
Medison is on a mission to ensure that every patient, regardless of where they live, has the opportunity to benefit from breakthrough therapies. For more information, please visit https://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 commercialize 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:
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- Australian Government Department of Health and Aged Care. Schedule of Pharmaceutical Benefits Summary of Changes Effective 1 October 2025 . Pharmaceutical Benefits Scheme (PBS) | New,amended and deleted
- Oxlumo Product Information, April 2025. Available at: www.medisonpharma.com.au/products/OxlumoPI
- Oxlumo Consumer Medicine Information, April 2025. Available at: www.medisonpharma.com.au/products/OxlumoCMI
- Garrelfs SF, Frishberg Y, Hulton SA et al. (2021). Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1. N Engl J Med. 384:1216-26. DOI: 10.1056/NEJMoa2021712
- Michael M, Groothoff JW, Shasha-Lavsky H. (2022). Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial. American Journal of Kidney Diseases, 81(2):145-155. DOI: 10.1053/j.ajkd.2022.05.012 ofthe phase 3 ILLUMINATE‑B trial. Pediatric Nephrology, 38:1075–1086. https://doi.org/10.1007/s00467-022-05684-1
- Hayes W, Sas D, Magen D et al. (2023). Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12‑month analysis