1 April 2026. Medison Pharma Australia Pty Ltd (“Medison”), alongside their partner Alnylam Pharmaceuticals, Inc. (“Alnylam”), is pleased to announce that from April 1st, 2026, GIVLAARI® (givosiran) is listed on the Pharmaceutical Benefits Scheme (PBS) for acute hepatic porphyria in adults and adolescents aged 12 years and older. 1,2,3
Acute hepatic porphyria (AHP) is a rare illness that runs in families.3 AHP presents more commonly in females between the ages of 20 and 40 years.4,5 It is caused by a defect in one of the proteins that make a molecule called haem in the liver.3 As a result there is a build-up of some of the substances that are used to produce haem, namely aminolevulinic acid (ALA) and porphobilinogen (PBG).3
Having too much ALA and PBG can injure nerves and other organs, and may cause serious attacks characterised by symptoms such as severe abdominal pain, nausea, muscle weakness and changes in mental functioning.3,4 Up to 8% of symptomatic patients experience recurrent attacks (≥4 attacks per year).4,5 Some patients may also experience symptoms, such as pain and nausea, between attacks. Longer-term complications that can be seen in people with AHP include high blood pressure, chronic kidney disease and liver disease.3
A/Prof Gayle Ross, Lead Clinician in the Multidisciplinary Porphyria Clinic at Royal Melbourne Hospital, said, “Acute Hepatic Porphyria (AHP) is a group of rare, complex, inherited metabolic disorders which causes a variety of impacts on multiple organs including the nerves and liver. It can result in poor quality of life, hospitalisations and rarely liver cancer and death. It is diagnosed by biochemical testing of porphyrins in blood, urine and faeces, and is confirmed with genetic testing. We are grateful to the Australian Government for making GIVLAARI® accessible on the PBS to patients with AHP.”
A/Prof. Peter Stewart, Chemical pathologist and Metabolic physician, past president of the Royal College of Pathologists of Australasia said, “Acute hepatic porphyria is a group of rare inherited disorders that result in attacks of severe abdominal pain, peripheral and autonomic neuropathy, metabolic and neuropsychiatric abnormalities, which can result in hospital admissions and, in some cases, can be fatal. A small number of these individuals can have multiple attacks requiring critical care management in a hospital. Individuals who have multiple attacks can develop continuing pain and nerve damage, hypertension and renal impairment. We welcome the PBS listing of GIVLAARI® in Australia as a treatment option for eligible patients suffering from AHP.”
GIVLAARI®(givosiran) is a medication used to treat acute hepatic porphyria and contains the active ingredient givosiran, a small interfering ribonucleic acid (siRNA)2. It works by lowering the amount of an enzyme called aminolevulinic acid synthase 1 (ALAS1), which controls how much aminolevulinic acid (ALA) and porphobilinogen (PBG) are made by the liver2. By lowering ALAS1, the liver makes less ALA and PBG.3
“The PBS listing of GIVLAARI® represents an advancement for people in Australia living with AHP. It underscores the importance of ensuring that innovative therapies reach patients without delay. At Medison, we are committed to strengthening global access pathways and working closely with our partners and health authorities to deliver treatments to patients in need,” said Victor Papamoniodis, Chief Commercial Officer at Medison.
Norton Oliveira, Senior Vice President and Head of Partner and Emerging Markets at Alnylam, said, “We are pleased to see this important step toward enabling access to GIVLAARI® for eligible patients living with acute hepatic porphyria in Australia. Through our collaboration with Medison, we remain committed to supporting those affected by this rare disease and helping address its significant burden.”
Adam Maguire, Country Manager, Australia at Medison, said, “There are limited treatment options for people living with AHP in Australia, and Medison is pleased with the PBS listing of GIVLAARI®. This milestone reflects our continued commitment to supporting the availability of innovative treatments within the Australian healthcare system.”
Each vial contains GIVLAARI® (givosiran) 1 mL of givosiran sodium equivalent to 189 mg givosiran.2
Indication: GIVLAARI® is used to treat acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older.
Dosage and Method of Administration: GIVLAARI® is administered as a subcutaneous (under the skin) injection once every month into the stomach area (abdomen), or in some cases, the upper arm or thigh. The GIVLAARI® injection should be given by a trained healthcare professional. The treating doctor will calculate the dose based on the patient’s actual body weight.
Contraindication: Do not use GIVLAARI® if the patient is allergic to givosiran or any of the other inactive ingredients (Sodium hydroxide, phosphoric acid, water for injections).
Warnings and Precautions: A doctor will determine if GIVLAARI® is a suitable treatment option for their patient with AHP. The doctor will consider other medical conditions and medicines, including pregnancy and breastfeeding. The safety and efficacy of GIVLAARI® in children aged < 12 years of age has not been established. Blood tests and monitoring of liver and kidney function may be required prior and during treatment.
Interactions: The patient should inform their doctor or pharmacist if they are taking any other medicines, including any medicines, vitamins or supplements they buy without a prescription from their pharmacy, supermarket or health food shop. Some medicines may interfere with GIVLAARI® and affect how it works. When using certain medicines, this medicine may prolong or increase their effect or change their side effects
Adverse Effects: All medicines can have side effects. Serious side effects include severe allergic reactions (swelling – mainly of the lips, tongue or throat which makes it difficult to swallow or breathe, breathing problems or wheezing, feeling dizzy or fainting, rash, hives, itching). Blood tests showing an increase in transaminases (liver enzymes),* an increase in creatinine (a substance removed from your body by your kidneys), or decrease in glomerular filtration rate* or an increase in homocysteine (a type of amino acid)*.
* These tests will be requested and interpreted by the doctor as part of routine monitoring before and during treatment.
For further information, patients should speak to their doctor and can refer to the GIVLAARI® (givosiran) Consumer Medicine Information available at https://www.medisonpharma.com.au/wp-content/uploads/Givlaari-AUS-CMI_approved_25Jul24.pdf
This media release is provided for informational purposes only. It is not intended to promote any prescription medicine to the general public or to replace professional medical advice.
PBS Information: GENERAL – General Schedule (Code GE) for patients with AHP
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. |
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For more information, please contact:
Medison Communications at corpcomms@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 the applicable regulatory requirements governing communication about prescription medicines to the general public. Medison Pharma gained the permission of all spokespeople to add their quotations before publication.
GIVLAARI® 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 March 2026, [AUS-ALN-NP-260001].
About Medison
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’s Partnership with Alnylam
Medison 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’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 April 2026 . Pharmaceutical Benefits Scheme (PBS) | New,amended and deleted
- GIVLAARI® (givosiran) Product Information, April 2025. Available at: https://www.medisonpharma.com.au/wp-content/uploads/Givlaari-AUS-PI_approved25Jul24.pdf
- GIVLAARI® (givosiran) Consumer Medicine Information, April 2025. Available at: https://www.medisonpharma.com.au/wp-content/uploads/Givlaari-AUS-CMI_approved_25Jul24.pdf
- Balwani, M et al. Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria. The New England Journal of Medicine. 2020. DOI: 10.1056/NEJMoa1913147
- Wang, B et al. Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study. Orphanet Journal of Rare Diseases. 2022. DOI: 10.1186/s13023-022-02463-x