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PNH

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

Novartis’ Fabhalta approved by FDA as first oral monotherapy for rare blood disease PNH

Novartis’ Fabhalta approved by FDA as first oral monotherapy for rare blood disease PNH

Victor Bultó, president US, Novartis, described the approval as an "extraordinary moment” for the PNH community. ... This new, effective oral medicine may mean that patients can reset their expectations of living with PNH,” he said.

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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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