Ultomiris shows strong interim results in Phase III IgA nephropathy trial

IgAN is a rare, progressive kidney disease driven by abnormal IgA immune complexes that deposit in the kidneys

Investigational therapy efzimfotase alfa shows promising Phase III results in rare bone disease

Efzimfotase alfa is an investigational enzyme replacement therapy designed to reduce injection volume

FDA nod to Koselugo for adults with Rare NF1 tumors, expanding life-changing treatment

EU approves Alexion’s Koselugo to treat adults with neurofibromatosis type 1 tumours

Approval based on pivotal KOMET Phase III trial demonstrating significant tumour reduction in adults with NF1

AstraZeneca’s Koselugo recommended for approval in the EU for Neurofibromatosis type 1

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

Voydeya approved in the EU as add-on treatment to ravulizumab

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

Ultomiris approved in the US for the treatment of adults with neuromyelitis optica spectrum disorder

First and only long-acting C5 complement inhibitor offers patients with AQP4 Ab+

AstraZeneca to acquire Amolyt Pharma

Acquisition to further expand the Alexion, AstraZeneca Rare Disease pipeline beyond complement inhibition

Ultomiris approved in the US for adults with generalised myasthenia gravis

Ultomiris showed early effect and lasting improvement in activities of daily living and has potential to reduce treatment burden with dosing every 8 weeks

AstraZeneca and Neurimmune to develop and commercialise NI006

Investigational human monoclonal antibody in Phase Ib development for the treatment of transthyretin amyloid cardiomyopathy, a systemic, progressive and fatal condition