EU regulators reject Santhera’s Raxone for DMD
September 18, 2017Swiss company to appeal regulators’ decision.
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EU regulators reject Santhera’s Raxone for DMD
https://pharmaphorum.com/news/eu-regulators-reject-santheras-raxone-dmd/
Rare diseases
FDA’s Gottlieb vows to close orphan drug loopholes
September 13, 2017
Alnylam halts trials of haemophilia drug after patient death
September 11, 2017Trials will resume once safety protocols have been reviewed.
Orchard’s gene therapy gains UK ‘promising innovative medicine’ status
August 23, 2017A UK biotech start-up has gained a sought after ‘promising innovative medicine’ (PIM) designation for its ultra-rare disease gene therapy.
Orphan drugs: overcoming HTA barriers to access
August 15, 2017How can orphan drug manufacturers demonstrate value and improve access to drugs?
Ionis to file rare disease drug after GSK declines option
August 14, 2017Firm turns down options for inotersen and IONIS-FB-LRX.
Alexion cuts ties with Moderna and re-focuses
July 27, 2017Alexion cuts collaborations with Moderna Therapeutics, Blueprint Medicines and Arbutus Biopharma
Amicus to file Fabry drug after FDA changes mind
July 12, 2017Regulator drops demand for further phase 3 trial.
