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orphan drugs

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

EMA hands UniQure orphan status first in Huntington's

EMA hands UniQure orphan status first in Huntington's

EMA hands UniQure orphan status first in Huntington's. The Dutch biotech expects to file the treatment for approval later this year. ... The European Medicines Agency (EMA) has granted UniQure’s investigational gene therapy an orphan medicinal product

Latest news

  • Shire separates out neuroscience as it considers its future Shire separates out neuroscience as it considers its future

    Historically dependent on just one ADHD treatment - Adderall -, neuroscience has dwindled in the face of Shire’s rising presence in orphan drugs and last year it accounted for less than a

  • Pharma is ‘getting lower returns on R&D’ Pharma is ‘getting lower returns on R&D’

    Pharma is ‘ getting lower returns on R&D’. And Deloitte says the number of drugs in late-stage trials tumbled by 16%. ... Deloitte also welcomes an increase in the number of approvals of new molecular entities, orphan drugs and break/fast-tracked

  • 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

  • Lucid launches rare and specialist disease agency Lucid launches rare and specialist disease agency

    According to analysts at Evaluate Pharma sales of orphan drugs are set to account for 20% of all prescription drug sales by 2020. ... The move mirrors an “industry-wide shift”towards focusing on rare and specialist disease, with revenues from orphan

  • EU’s 10-year paediatric meds report - not bad, but can do better EU’s 10-year paediatric meds report - not bad, but can do better

    These include “off-label use of products designed for adults, un-adapted rewards for innovation, and the lack of use of the Orphan Drugs Regulation for paediatric indications”, she said. ... I am all the more afraid that the ongoing incentives review

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Latest Intelligence

  • Drug R&D

    Healthcare terms - Drug R&D.

  • The sound of silence puts the squeeze on generics and biosimilars The sound of silence puts the squeeze on generics and biosimilars

    drugs - from Adalimumab to Rituximab - go off-patent and boost savings across eight European nations to 33.4bn over the next three years. ... What is surprising is that the whole of Europe is deeply concerned about the price of new medicines and how it

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

    Altogether 39 orphan drugs were approved in the US during 2016 and 14 in the EU (a mix of NME, BLA, NAS or old substances with a new use for an ... We expect the strong overall trend for more orphan drugs submitted to and approved by the FDA and the EMA

  • Shoring up the framework Shoring up the framework

    This first document, set out in a Q&A format, covers information on company location, with respect to centralised procedures and certain other activities, including the location of orphan designation holders ... Marketing authorisation holders for

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

    Under fire. Some observers feel that Germany and France provide relatively wide access to orphan drugs, certainly compared to England. ... Numbers game. The number of orphan drugs actually being approved in Europe is hard to quantify.

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Latest appointments

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Latest from PMHub

  • Ashfield to present at the Orphan Drugs for Rare Diseases Summit

    Ashfield has a breadth of experience with orphan drugs and we are passionate about helping to improve the lives of people living with rare diseases. ... We are delighted to be sponsoring and presenting at the ‘ Orphan Drugs for Rare Diseases’ event 22

  • 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. ... Firstly, manufacturers of orphan drugs are awarded longer market exclusivity, reduced

  • Finding the familiar in Rare Disease

    Finding the familiar in Rare Disease. Inspired by World Rare Disease Day, WE Communication's Hugh Adams covers some of the challenges faced when considering Rare Disease communications. Estimates suggest that a so-called rare disease affects one in

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

    Market research with many of the industry leaders involved with the development of orphan drugs has indicated the main influential drivers to pursuing an orphan drug approval as the reasons shown ... The necessary infrastructure, understanding of orphan

  • Magic of Lucid targets rare and specialist diseases

    This move mirrors an industry-wide shift towards focusing on rare and specialist diseases, with revenues from orphan drugs outpacing sales of mainstream drugs for the last decade, a trend that’ ... According to the Orphan Drug Report (2015), sales of

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