All Orphan Drugs articles
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NewsIpsen strikes €1bn deal to acquire French biotech ImCheck Therapeutics
The move will boost its oncology pipeline, adding a potential first-in-class anti-BTN3A monoclonal antibody for acute myeloid leukaemia.
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NewsNovartis scores Phase III wins for two ianalumab indications
Late-stage trials in Sjögren’s disease and primary immune thrombocytopenia met primary endpoints.
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NewsCHMP meeting highlights - May 2025
The EMA’s committee has granted positive opinions for ten new treatments, including medicines for oncology indications and rare diseases.
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ArticleBiosimilar medicines: the intersection of access, affordability, and innovation
In this article, Julie Maréchal-Jamil, Director, Biosimilar Policy & Science, Medicines for Europe, explores the strategies needed at European and national levels to secure the benefits of biosimilar medicines in an evolving landscape to deliver a pipeline for these drugs by 2030.
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NewsEuropean Commission licenses first treatment for acanthamoeba keratitis
Following its European approval, the small molecule treatment is set to become the standard of care for patients with the ultra-rare disease.
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NewsOligonucleotide ALS therapy approved in EU
The European Commission (EC) has granted Biogen its third rare disease treatment approval in the European Union.
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ArticleWhy patient-centricity is essential to accelerate rare disease clinical research
There is an urgent need for novel treatment options for people living with rare diseases, but carrying out clinical research in this field is notoriously difficult. As Clinical Trials Day approaches, Cezary Statuch, Head of Medical Affairs International and Japan at Alexion Pharmaceuticals, highlights how adopting patient-centric approaches can help ...
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ArticleEU pharmaceutical legislation revisions: what are the implications for biopharma?
Anne Dhulesia and Sean Dyson, Partners at L.E.K. Consulting, discuss the proposed revisions to EU pharma legislation and potential implications for biopharma companies operating in Europe.
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NewsCHMP meeting highlights – May 2023
The Committee for Medicinal Products for Human Use (CHMP) recommended two new medicines for approval in its May meeting, including a neurosteroid for epileptic seizures.
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NewsEMA’s human medicines committee (CHMP) meeting highlights - February 2023
In its latest meeting, the Committee for Medicinal Products for Human Use (CHMP) recommended eight new medicines for approval, including an enzyme replacement therapy for a rare disease.