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PNH

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

AZ’s PNH therapy Ultomiris wins EU backing to expand use for children

AZ’s PNH therapy Ultomiris wins EU backing to expand use for children

PNH is a rare blood condition where red blood cells are destroyed and can result in blood clots and impaired bone marrow function. ... who are clinically stable after treatment with Alexion’s older PNH drug Soliris (eculizumab).

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  • Orphan drugs and breakthrough therapies drive Cortellis Drugs to Watch 2019 Orphan drugs and breakthrough therapies drive Cortellis Drugs to Watch 2019

    It is a next- generation follow-on to Soliris, Alexion’s current PNH blockbuster – also a humanised monoclonal antibody – with more convenient dosing. ... Ultomiris received approval as an Orphan Drug from the FDA for adult patients with PNH in

  • Drug launches to watch in 2019 Drug launches to watch in 2019

    Alexion is bracing for the patent expiry of its blockbuster Soliris (eculizumab), used to treat paroxysmal nocturnal haemoglobinuria (PNH) and myasthenia gravis, which hit revenues of around $3bn in 2018. ... The follow-up, Ultomiris ALXN1210, is a second

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