All Rare diseases articles
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NewsServier deepens rare oncology commitment with $2.5bn Day One acquisition
The deal aligns with the biopharma company’s goal of expanding its oncology pipeline by the end of the decade.
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NewsBiogen/Stoke’s zorevunersen hailed as potential first disease-modifying drug for rare epilepsy
Clinical readout for Dravet syndrome trial part of the collaboration the companies signed in 2025.
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NewsBlueprint’s rare disease drug Ayvakit shows long-term benefit
D816V-targeted therapy provided robust symptom control in patients with indolent systemic mastocytosis, supporting its use as a long-term treatment.
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NewsFDA drafts new ultra-rare disease therapy guidance
Agency’s planned framework is the first to support the approval process for new ultra-rare disease treatments.
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NewsAmgen wins new European approval for Uplizna in generalised myasthenia gravis
The EC decision clears a new first-in-class approach to manage the rare autoimmune condition.
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NewsNMD Pharma will ‘accelerate’ rare disease drug ignaseclant despite endpoint miss
But phase II data of the small molecule inhibitor for Charcot-Marie-Tooth disease Types 1 and 2 did show it meeting secondary endpoints for functional improvements.
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NewsModerna and Recordati collaborate on rare disease mRNA therapy
License agreement set to advance mRNA-3927, a potential disease modifying therapy for the rare metabolic disorder propionic acidemia.
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NewsSentynl secures US-first approval for rare Menkes disease
The US biopharma company can now offer a new treatment option for eligible paediatric patients with the genetic disease.
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AstraZeneca furthers US manufacturing commitment with $2bn Maryland investment
The move is part of biopharma's $50 billion pledge to US medicines manufacturing and R&D.
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NewsMHRA set to overhaul the UK’s rare disease drug regulatory pathway
The medicines regulator will aim to take a more flexible licensing approach for the research and manufacture of rare disease therapies in the UK.
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NewsAlexion wins EU label expansion for kinase inhibitor Koselugo
Approval provides continuity of care into adulthood for patients with neurofibromatosis type 1 (NF1) using Koselugo (selumetinib) to manage symptoms.
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NewsKeytruda post-surgery could halt return of distant tumours in rare skin cancer
Merck & Co’s anti-PD-1 immunotherapy showed evidence at ESMO of its ability to prevent cancer recurrence in Merkel cell carcinoma.
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NewsOne-time gene therapy could “transform” Huntington’s treatment landscape
uniQure’s positive topline data “are the most convincing in the field to date” and indicate the gene therapy’s potential disease-modifying effects.
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NewsCPHI Pharma Awards 2025 finalists announced
Winners of this year's awards will be revealed during the opening of CPHI Frankfurt 2025.
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FDA to modernise review process for developers of ultra-rare diseases
The regulator’s new principles aim to ease the pathway to regulatory approval for rare disease drug developers in the US.
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NewsMerck’s SpringWorks Therapeutics gains European approval for Ogsiveo
The drug is the first treatment for desmoid tumours to be approved in the region.
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Amid FDA turmoil Sarepta’s gene therapy worries begin to ease
But the impact of George Tidmarsh’s CBER appointment after Vinay Prasad’s abrupt resignation remains to be seen.
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NewsEuropean Council announces progress on pharmaceutical package adoption
The new EU rules for medicines aims to strengthen EU pharmaceutical competitiveness and reduce the regulatory burden.
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ArticlePreparing for a new era in the rare disease sector
To mark Rare Disease Day 2025, EPR interviewed Christina Gkousgkouni, Head of Rare Diseases for Central South Europe, Sanofi.
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NewsNovo Nordisk investing in rare disease with major new manufacturing site
A total of DKK 8.5 billion will fund the new production facility in Odense, Denmark, which is set to facilitate the manufacture of medicines for rare diseases.