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  • NICE reject two treatments for fatal, ultra-rare disease – hATTR pharmafile
    December 14, 2018
    The NHS’ cost effectiveness body NICE has rejected two treatments for the rare inherited condition, hereditary transthyretin-related amyliodsis (hATTR), over concerns as to whether the drugs showed long term benefits.
  • FDA delays decision on inotersen, handing lead to Alnylam fiercebiotech
    May 07, 2018
    The FDA has delayed the PDUFA date for Akcea’s hereditary TTR amyloidosis drug inotersen, handing first-mover advantage to Alnylam. Akcea suffered the setback after the FDA asked for more time to review its response to requests for information ...

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