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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NewsUCB wins CHMP favour for first thymidine kinase 2 deficiency treatment
If approved by the European Commission, Kygevvi would provide a new treatment option for eligible patients with the ultra-rare mitochondrial disease in Europe.
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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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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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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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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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NewsEli Lilly to lead oligonucleotide therapy development in new collaboration
The partnership between Eli Lilly and Company and Creyon Bio will focus on producing safer, more effective RNA-targeted oligonucleotide therapies with the use of AI technology.
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NewsCHMP meeting highlights – February 2025
The Committee for Medicinal Products for Human Use (CHMP) recommended approval of four new medicines plus the extension of therapeutic indications for 16 medicines.
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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.
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NewsNovartis to advance novel Huntington's disease therapy
As part of the agreement with Novartis Pharmaceuticals, PTC will receive an initial $1 billion to develop the potential first-in-class oral therapy.
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NewsInnovating LNP design to improve mRNA therapeutics
The new approach involving refinement and optimisation of ionisable lipids could accelerate the development of mRNA therapies, research suggests.
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NewsSanofi collaboration to advance innovative radioligand therapies
The new partnership between Sanofi and Orano Med, delivered via a new entity valued at nearly $2 billion, will focus on progressing lead-212 based targeted alpha therapies for oncology indications.
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NewsAAV gene therapy manufacturing gets £1.4m boost
The funding aims to reduce the costs of AAV production for gene therapies, helping to accelerate treatment access for patients with rare diseases.
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ArticleEnzyme replacement therapy: improving outcomes in rare disease
In this article, Dr Alaa Hamed, Global Head of Medical Affairs for Rare Disease at Sanofi, shares why the company’s enzyme replacement therapy Xenpozyme® (olipudase alfa) has promise for the rare genetic disease acid sphingomyelinase deficiency (ASMD).