Approval based on pivotal KOMET Phase III trial demonstrating significant tumour reduction in adults with NF1
The positive opinion is based on results from KOMET, the largest and only placebo-controlled, double-blind global Phase III trial in this patient population
ALPHA Phase III trial showed first-in-class, oral, Factor D inhibitor as add-on to Ultomiris or Soliris improved haemoglobin levels and reduced anaemia and fatigue
First and only long-acting C5 complement inhibitor offers patients with AQP4 Ab+
Acquisition to further expand the Alexion, AstraZeneca Rare Disease pipeline beyond complement inhibition
Ultomiris showed early effect and lasting improvement in activities of daily living and has potential to reduce treatment burden with dosing every 8 weeks
Investigational human monoclonal antibody in Phase Ib development for the treatment of transthyretin amyloid cardiomyopathy, a systemic, progressive and fatal condition
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