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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.

Orphan drugs from Biogen and Amicus win UK Prix Galien

Orphan drugs from Biogen and Amicus win UK Prix Galien

There was a much warmer welcome at the awards, hosted by ValueBase, for the pharma and biotech companies with products on the shortlist, which was dominated by orphan medicines. ... All bar one of the finalists were medicines for orphan conditions, and

Latest news

  • Malthus’ orphans Malthus’ orphans

    Shire. The attractiveness of rare diseases, orphan drugs and other ‘ultra niche’ strategies is their high relative profitability; governments tolerate high cost per patient because the patients are few and often ... By and large, rare diseases and

  • The year of the blockbuster The year of the blockbuster

    The diseases targeted by the report’s projected market entrants include type 2 diabetes, endometriosis, childhood epilepsy, haemophilia, HIV, migraine, opioid addiction and shingles. ... Five of the 12 drugs  we identified are first-in-class  and

  • Drugs for rare diseases will help propel pharma sales to $1.2trn by 2024; report Drugs for rare diseases will help propel pharma sales to $1.2trn by 2024; report

    That assertion ties in with its new report on worldwide prescription drug sales, which says that drugs for orphan diseases will outperform the overall pharma market, almost doubling to reach a ... That will also be a factor for orphan drugs, with recent

  • Celgene bulks up pipeline again with $2.1bn Prothena deal Celgene bulks up pipeline again with $2.1bn Prothena deal

    smaller $101m collaboration with Vividion Therapeutics that also covers cancer and inflammatory diseases targets. ... Our collaboration leverages each company’s core expertise in protein homeostasis and protein clearance to target proteins that are the

  • Duchenne UK and pharma partner pilot data-sharing HTA approach Duchenne UK and pharma partner pilot data-sharing HTA approach

    The pharma partners - Roche, Sarepta Therapeutics, Solid Biosciences, Summit and Wave Life Sciences - all have an interest in DMD as well as other rare and orphan diseases that can have difficulties ... These delays are worsened for diseases as there

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Latest Intelligence

  • Europe vs the US: New drug product approvals Europe vs the US: New drug product approvals

    During 2016, special approval procedures like conditional and accelerated approval pathways were issued for seven of the new approvals in the EU, the majority in oncology and for orphan diseases. ... We expect the strong overall trend for more orphan

  • Rare diseases: meeting the challenge Rare diseases: meeting the challenge

    However, fewer than 1, 000 diseases ‘benefit from even minimal amounts of scientific knowledge’, the EMA says. ... Numbers game. The number of orphan drugs actually being approved in Europe is hard to quantify.

  • Deal Watch February 2017 Deal Watch February 2017

    IMMU-132 has FDA Breakthrough Therapy designation for the treatment of patients with TNBC who have failed prior therapies for metastatic disease and also has Fast Track status and orphan designation ... The amendment gives Vifor the rights to

  • A rare talent A rare talent

    Shire’s Sebastian Stachowiak on dealing with change and working for a fit-for-purpose orphan drug environment. ... A rare challenge. That knowledge is currently focused on bringing new treatments to market for rare diseases and working to ensure the

  • Mastering the rare disease challenge Mastering the rare disease challenge

    Identifying expertise and customising communication in orphan drug marketing. Orphan drugs are few and far between. ... Clinicians often have little or no concept of how diseases like sarcoidosis or systemic sclerosis present themselves and have

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