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Orphan drugs and rare diseases

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

Lucid picks up rare disease and orphan drugs trophy at PMEA 2017

Lucid picks up rare disease and orphan drugs trophy at PMEA 2017

Lucid picks up rare disease and orphan drugs trophy at PMEA 2017. ... Judges praised the AbbVie project for its impressive reach. Lucid picked up the award for Excellence in Rare Diseases and Orphan Drugs at this year’s PMEA Awards, which were hosted

Latest news

  • Celgene scoops Socially Responsible Initiative prize at PMEA 2016 Celgene scoops Socially Responsible Initiative prize at PMEA 2016

    The objectives were laudable and the implementation made a big difference to many children's lives.”. ... Celgene was also a finalist with '75 Not Out' in the Rare Diseases and Orphan Drugs group.

  • Shire completes $32bn Baxalta merger Shire completes $32bn Baxalta merger

    Shire has finalised its $32bn merger with Baxalta, creating one of the world's leading firms specialising in rare diseases and conditions. ... Shire now has over 50 rare diseases programmes in various stages of clinical development and, together with its

  • Novel drugs and rare diseases drive US prescription drug spend Novel drugs and rare diseases drive US prescription drug spend

    Novel drugs and rare diseases drive US prescription drug spend. But increased competition and cost-cutting measures forced price concessions. ... It notes that there were 43 new active substances launched in the US last year, with a high proportion of

  • Wakix backed for European approval in narcolepsy Wakix backed for European approval in narcolepsy

    Wakix backed for European approval in narcolepsy. Bioprojet Pharma’ s orphan drug given a positive opinion by CHMP. ... Narcolepsy is a rare, long-term sleep disorder, which affects the brain's ability to regulate the normal sleep-wake cycle.

  • FDA grants Tocagen’s glioblastoma drug orphan status FDA grants Tocagen’s glioblastoma drug orphan status

    FDA grants Tocagen’ s glioblastoma drug orphan status. Puts the brain cancer gene therapy in-line for development incentives. ... We believe the FDA's granting of both orphan drug and fast track designations to Toca 511 &Toca FC will enable us to more

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Latest Intelligence

  • A rare talent A rare talent

    Change has also been the order of the day at Shire, with the Dublin-headquartered company taking strides towards future growth over the last couple of years, with rare diseases and ... A rare challenge. That knowledge is currently focused on bringing new

  • Reaching new heights Reaching new heights

    for rare diseases and technology-enabled changes in care that can harness big data to better inform decisions, help drive patient behaviour changes and improve outcomes. ... biologics. Patients will have greater access to breakthrough therapies and

  • Applying real-world evidence to orphan drugs Applying real-world evidence to orphan drugs

    This is particularly challenging in the area of rare diseases, in which there have been substantial advances, largely due to our increased knowledge of the genomic and biological makeup of the ... In Europe, the EU Committee of Experts on Rare Diseases

  • Another lost decade for research? Another lost decade for research?

    Another lost decade for research? What does the patent landscape look like for antibiotics, vaccines and orphan drugs? ... The annual number of patent families filed in the field of rare diseases is far higher than for vaccines or antibiotics.

  • The distressing impact of rare diseases The distressing impact of rare diseases

    What role can pharma play? The pharmaceutical industry is undergoing a transformative approach to rare diseases, as orphan drugs address a high unmet medical need and have been proven to be ... This article was originally published in the PME supplement

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Approximately 1 fully matching, plus 13 partially matching documents found.

Latest from PMHub

  • Pharma Market Research Report - Predictions for 2016

    2016 will continue to see an increase in the number of requests for research projects investigating the market opportunity for rare disease and orphan drugs. ... Last year the FDA approved 48 drugs in rare diseases, including both novel and repurposed

  • Finding the familiar in Rare Disease

    There is a continuing struggle for people affected by rare diseases to build a confident platform, to manage their condition and not to be defined by it. ... Where rare diseases seem to differ is how diagnoses are communicated, conveyed in broad terms

  • Lucid’s magic touch wins them two coveted trophies at the PMEA

    The company won its first award of the night in the category “ Excellence in Rare Diseases and Orphan Drugs” for the EIDON programme, which focuses on establishing specialist care for patients ... OptiMiSe is aimed at multiple sclerosis specialist

  • Can rare diseases be a viable option for the pharma industry?

    Moving into rare diseases will provide an opportunity for big pharma to diversify and begin to move away from developing drugs for the mass market indications. ... With a significantly reduced investment profile for product development and marketing

  • Lucid’s magic touch wins them two coveted trophies at the PMEA

    The company won its first award of the night in the category “ Excellence in Rare Diseases and Orphan Drugs” for the EIDON programme, which focuses on establishing specialist care for patients ... OptiMiSe is aimed at multiple sclerosis specialist

More from PMHub
Approximately 2 fully matching, plus 13 partially matching documents found.

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