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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 dominate FDA's record-breaking year

Orphan drugs dominate FDA's record-breaking year

drugs, driven on by new pharma and biotech science, and the FDA's determination to accelerate its review times. ... are treated.” Ultomiris need only be dosed every eight weeks, while current drugs for PNH have to be given every two weeks.

Latest news

  • Better together when buying medicines? Taking stock of the BeNeLuxA initiative Better together when buying medicines? Taking stock of the BeNeLuxA initiative

    Many more documents have been produced on HTA. While there have not been joint HTAs for all six of the drugs looked at, there has been a joint HTA (and it ... It’s not just price that was subject to negotiation. In a way that has become almost business

  • PMEA 2018: celebrating excellence in improving patient outcomes PMEA 2018: celebrating excellence in improving patient outcomes

    AbbVie also took home two other awards, Excellence in Healthcare Professional Education Support Programmes (with support from Sciterion) and Excellence in Rare Diseases and Orphan Drugs (with support from Lucid). ... Meanwhile, for AbbVie’s and

  • 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

  • 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.

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  • 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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