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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.

Orphan drugs from Biogen and Amicus win UK Prix Galien

Orphan drugs from Biogen and Amicus win UK Prix Galien

Orphan drugs from Biogen and Amicus win UK Prix Galien. Amicus’Galafold and Biogen’s Spinraza have won the two top prizes at the UK Prix Galien, the biennial awards which ... There was a much warmer welcome at the awards, hosted by ValueBase, for the

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  • Malthus’ orphans Malthus’ orphans

    By and large, rare diseases and orphan drugs don’t lend themselves to the former or the latter. ... Life sciences companies that see orphan drugs and other diseases as their future might do well to consider the threat of Malthus’trap.

  • The year of the blockbuster The year of the blockbuster

    Five of the 12 drugs  we identified are first-in-class  and four are indicated for  orphan diseases. ... from two similar drugs targeting CGRP - from Eli Lilly and Teva - is not far behind.

  • Orphan drugs dominate UK Prix Galien shortlist, medtech debuts Orphan drugs dominate UK Prix Galien shortlist, medtech debuts

    Orphan drugs dominate UK Prix Galien shortlist, medtech debuts. Seven treatments for rare conditions have been selected for the final. ... The shortlist for one of the UK’s most prestigious pharma industry prizes has been announced, and illustrates the

  • 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

    This highlights the industry’s continued move to address small groups of neglected patients with high unmet need and to benefit from traditionally reduced payer scrutiny on orphan drugs, as well ... That will also be a factor for orphan drugs, with

  • 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

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  • 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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