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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
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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Novartis’ iptacopan scores positive results in rare blood disorder PNH
PNH is a rare and life-threatening blood disorder with often debilitating symptoms,” said John Tsai, head global drug Development and chief medical officer, Novartis. ... We are excited to continue to explore the potential of iptacopan as new
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NICE recommends Ultomiris for NHS use in patients with rare blood disorders
PNH is a rare blood condition, wherein red blood cells are destroyed which can result in blood clots and impaired bone marrow function. ... Ultomiris has also been found to cause fewer episodes of breakthrough haemolysis – a symptom of PNH – thereby
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FDA approves Apellis’ Empaveli for rare blood disease
PNH, an autoimmune disease characterised by the destruction of red blood cells and anaemia. ... Apellis is also conducting a study of Empaveli in treatment-naïve PNH patients – meaning they have not previously received a complement inhibitor within
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Alexion and NICE agree confidential discount for Ultomiris use on the NHS
NICE said clinical evidence shows Ultomiris is as effective and safe as Alexion's older PNH drug Soliris. ... stable after receiving Alexion’s older PNH drug Soliris (eculizumab) for six months or more.
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Novartis receives FDA breakthrough therapy designation for rare disease med
Swiss pharma company Novartis has received a breakthrough therapy designation (BTD) for its experimental medication iptacopan for the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH). ... standard of care anti-complement treatment, as well
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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
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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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