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orphan diseases

This page shows the latest orphan diseases news and features for those working in and with pharma, biotech and healthcare.

EC approves Atara and Pierre Fabre’s Ebvallo as monotherapy for Epstein-Barr patients

EC approves Atara and Pierre Fabre’s Ebvallo as monotherapy for Epstein-Barr patients

We are proud and excited to bring this innovative therapy to the marketplace, which will reinforce Pierre Fabre’s portfolio in oncology, haematology and rare diseases.”. ... Ebvallo has orphan designation in Europe, which is reserved for therapies

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  • Listen, learn and lift off Listen, learn and lift off

    The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are expecting a busy year for approvals, with frantic activity in orphan diseases as gene and cell therapy ... That has completely changed and we’re now launching

  • Piloting a route to the biosimilars opportunities in Europe Piloting a route to the biosimilars opportunities in Europe

    Biological medicines represent a major advance in the treatment of serious pathological conditions such as cancer, neurodegenerative and autoimmune diseases, in the form of biosimilars. ... For orphan designations (medicine for rare diseases),

  • Assessing the impact of disease prevalence on access and pricing of rare disease drugs in England Assessing the impact of disease prevalence on access and pricing of rare disease drugs in England

    Comparison of orphan drug assessments from NICE versus the NHS. The question of how to adequately capture the holistic value of orphan drugs (ODs) for rare diseases is the subject of ... to drugs targeting ‘very rare’ diseases (typically ultra-orphan

  • Biopharma and orphan drugs Biopharma and orphan drugs

    Rare disease regulations. Twenty one of the 48 novel drugs approved in the US were for rare or orphan diseases – those that affect fewer than 200, 000 citizens. ... Clinical complications. Given the rarity of orphan diseases, one of the greatest

  • Industry needs an aligned position on the future of the EU OMP Regulation Industry needs an aligned position on the future of the EU OMP Regulation

    The Pharmaceutical Strategy is likely to include innovation-boosting measures and plans to revise the EU Regulation on Orphan Medicinal Products (OMP Regulation). ... of orphan diseases).

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  • Rare and Orphan Diseases Rare and Orphan Diseases

    1. It’s also predicted that by 2026 orphan drugs will make up almost 1/3 of the global drug pipeline’s value. ... Hadjivasiliou, A. and Senior, M. ‘Evaluate Orphan Drug Report 2022’. Available at: (Date accessed: January 2023).

  • Rare Diseases - not as rare as you think Rare Diseases - not as rare as you think

    There are more than 7000 rare diseases affecting an estimated 3.5 million people in the UK. ... The European Medicines Agency (EMA) supports the development and authorisation of medicines for rare diseases (often referred to as ‘orphan’ diseases)

  • Onyx Health Joins Forces with Rare Disease Specialists Actigen Onyx Health Joins Forces with Rare Disease Specialists Actigen

    Actigen's work focuses on identifying and developing biological medicines for rare diseases with unmet medical needs. ... At Actigen, our mission is to improve the lives of individuals with rare diseases, and we hope that GNR-055 will be the start of

  • How to Simplify Genetics & Raise Awareness Around Orphan Diseases

    Gail Ouellette, President and Scientific Director of Regroupement Québécois des Maladies Orphelines ( RQMO)/Quebec Coalition of Orphan Diseases and Creator of the “ Genetics Simply" website, shares her thoughts on the barriers ... faced by rare

  • Lucid Group has four programmes shortlisted for PMEA 2019

    Category: Excellence in Innovation. DermDialogue: Bringing innovation to MSL‐led education. Excellence in Rare Diseases and Orphan Drugs.

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