Chief executive Jon Pilcher said the approval was especially meaningful given the lack of an approved Rett syndrome treatment in Europe to date.
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Latest News
Australia cannot deliver meaningful change until the levers of power are rebalanced
Any assessment of Australia’s use of health technology assessment requires an historical lens, at the very least for context, but also for learnings. The system's history makes it predictable. It is how we know what comes next.
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Latest News Australian company secures European approval for rare disease treatment August 26, 2026
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Latest News Professor Brandon Wainwright appointed to lead QIMR Berghofer August 25, 2026QIMR Berghofer, one of Australia's leading medical research institutions, has appointed internationally recognised geneticist Professor Brandon Wainwright AM as its next Director and Chief Executive Officer.
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Latest News Australian study finds maternal RSV vaccination offers strong protection for infants August 25, 2026Dr Krishan Thiru, Medical Director for Pfizer Australia and New Zealand, said the results provide important local evidence at a time when infants face their greatest risk of severe RSV disease.
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Latest News Insurers warn cut could hit Australians most likely to need cancer care August 25, 2026The organisation has pointed to the potential impact on cancer treatment and care, highlighting that the policyholders impacted by the reduction are more likely than any other age group to access cancer treatment.
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Australian Biotech Acrux brings experienced clinical leader aboard to advance testosterone program August 24, 2026Acrux has appointed physician and biopharmaceutical executive Dr Jolanta Airey as its chief medical officer, strengthening its leadership team as it prepares to advance its female testosterone treatment toward Phase 3 clinical trials in the United States.
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Latest News These rarely stated realities should be discussed in the open more often August 24, 2026At the congress, an experienced PBAC member offered a rare glimpse inside Australia’s medicines-reimbursement institution. It was imperfect in its facts, but critically important and worth welcoming, particularly the comments about patient input and the 'reality' of the 'emotive pull' of cancer.
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Latest News July recommendations across oncology, rare disease and chronic care August 24, 2026At its July meeting, the committee backed multiple submissions but rejected or deferred others for the regularly cited reasons, including pricing.
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Latest News The claim Australia cannot invest more in new medicines fails very quickly August 24, 2026The Government's own projections for one sub-program of the National Disability Insurance Scheme show that Australia can afford a more ambitious investment in new medicines. It is a choice.
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Latest News Opportunity #5 - From the hardest thing to secure to the easiest thing to get right August 21, 2026As the medicines industry prepares to negotiate new agreements with the Commonwealth, the one thing it must get right is the easiest to understand. That is, power dependence.
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Podcast The 'Dispatched' Podcast - Series 5. Ep.29 August 21, 2026The opportunities available in the pending negotiation of new industry agreements and the selective discrimination against some patients and patient groups by an outdated and unaccountable institution.
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Latest News Personalised mRNA cancer therapy clears Phase 3 milestone in resected melanoma August 21, 2026Professor Georgina Long, the study's principal investigator and medical director of Melanoma Institute Australia, said the findings could signal a shift in post-surgical melanoma treatment.
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Latest News Biogen bolsters leadership ranks as it sharpens focus on patient access and innovation August 21, 2026Biogen Australia and New Zealand has bolstered its leadership team by appointing Sarah Payne as Head of Medical ANZ and Elias Toomeh as Head of Value and Access.
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Latest News IDT Australia targets profitability after reporting sharply narrowing loss August 21, 2026Executive Chair Mark Simari said 2026 marked a turning point for the company, with the improved financial performance and manufacturing investment positioning IDT for its next stage of growth.
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Advent moves to acquire majority stake in NZCR Group August 21, 2026Global private equity firm Advent has agreed to acquire a majority shareholding in New Zealand Clinical Research Group, marking a significant new investment in the Australian and New Zealand clinical-trials sector.
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Latest News Opportunity #4 - Scope creep demands stronger checks and balances August 19, 2026Opportunity number four for industry in the pending negotiation of new agreements may be the hardest to achieve, but it also offers the greatest potential to put Australia’s medicines decision-making framework on track.
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Latest News Rare autoimmune diseases impose a $1.2 billion annual burden on Australians August 20, 2026Patients Australia chair Michele Robbins said the report demonstrated why the assessment of rare diseases needed to take greater account of the patient, family and societal impact of delayed diagnosis and restricted access to effective treatment.
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Latest News New Zealand researchers honoured for work tackling superbugs and blood cancer August 20, 2026Two New Zealand scientists working at very different points of the medicines-development pipeline will be recognised tonight at Medicines New Zealand’s 2026 Value of Medicines Awards.
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Latest News Recce advances pivotal diabetic foot infection trial with active antibiotic comparator arm August 20, 2026Recce Pharmaceuticals has secured Australian ethics approval to expand its pivotal Phase 3 trial of RECCE 327 Topical Gel by adding a head-to-head study against antibiotics currently used to treat diabetic foot infections.
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Latest News Opportunity #3 - finally deliver on the repeated claims of patient-centricity August 19, 2026The continued exclusion of patients from these discussions, characterised by closed-shop secrecy, is inconsistent with the proper administration of a public health program. Joint presentations at the start and end of the process are not enough.
Australian Biotech
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Noxopharm pushes Sofra platform towards cancer proof of concept
August 26, 2026 -
Neuren’s first dividend signals a new phase built on DAYBUE royalties
August 26, 2026 -
Starpharma narrows loss as Genentech deal lifts revenue and new funding extends runway
August 26, 2026 -
Genetic Signatures and Microba explore merger to build infectious disease diagnostics group
August 26, 2026 -
Australian company secures European approval for rare disease treatment
August 26, 2026
Other Health
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Rare autoimmune diseases impose a $1.2 billion annual burden on Australians
August 20, 2026 -
New partnership aims to create national blueprint for earlier Alzheimer’s diagnosis and care
July 31, 2026 -
Melbourne study links higher tumour radiation dose to longer progression-free survival
July 10, 2026 -
Tax deal delays NDIS overhaul and reopens scrutiny with inquiry extension
June 24, 2026 -
NDIS fraud is so extensive that 'prosecuting our way out of it' is now impractical
April 24, 2026
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