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Results For "AstraZeneca-Rare-Disease"

9 News Found

AstraZeneca reports major Phase III breakthrough in IgA kidney disease trial
Clinical Trials | June 09, 2026

AstraZeneca reports major Phase III breakthrough in IgA kidney disease trial

At 34 weeks, Ultomiris achieved a 43.4% placebo-adjusted reduction in 24-hour urine protein creatinine ratio (UPCR) versus placebo, meeting the study’s key interim endpoint


AstraZeneca reports mixed Phase III results for anselamimab in rare cardiac amyloidosis trial
Clinical Trials | May 30, 2026

AstraZeneca reports mixed Phase III results for anselamimab in rare cardiac amyloidosis trial

The programme evaluated anselamimab as a first-line add-on to standard plasma cell dyscrasia therapy in patients with advanced cardiac AL amyloidosis


Ultomiris shows strong interim results in Phase III IgA nephropathy trial
Clinical Trials | April 22, 2026

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


Alexion to showcase broad neurology pipeline at AAN 2026 with 20 presentations
R&D | April 14, 2026

Alexion to showcase broad neurology pipeline at AAN 2026 with 20 presentations

The programme features headline data from pivotal Phase III trials, including PREVAIL in gMG, KOMET in NF1-PN, and CHAMPION-NMOSD, alongside real-world evidence supporting established therapies


Investigational therapy efzimfotase alfa shows promising Phase III results in rare bone disease
Clinical Trials | April 01, 2026

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


EU approves Alexion’s Koselugo to treat adults with neurofibromatosis type 1 tumours
Clinical Trials | October 31, 2025

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


Alexion highlights breakthrough C5 inhibition advances in gMG at 2025 AANEM and MGFA scientific sessions
Clinical Trials | October 31, 2025

Alexion highlights breakthrough C5 inhibition advances in gMG at 2025 AANEM and MGFA scientific sessions

New Phase III data on gefurulimab highlight potential as a self-administered treatment option; real-world evidence reinforces clinical benefits of Ultomiris and Soliris


AstraZeneca to acquire Amolyt Pharma
News | March 18, 2024

AstraZeneca to acquire Amolyt Pharma

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