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Mid Specialty · Rare Disease

Alexion (AZ Rare) — Rare Disease

Alexion (AZ Rare) across Rare Disease: the full competitive set, and the 6 AXLRx reports mapping the competitive, patient-flow and payer picture — each scoped to a single asset and cited to a live source.

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AXLRx reports featuring Alexion (AZ Rare) — Rare Disease

CI · Rare Disease · CI Team · Launch Lead US PNH Competitive Intelligence Iptacopan oral pivot versus the anti-C5 IV class. Orphan-drug exclusion from IRA negotiation (US), NICE HST (UK) and SFDA lag (GCC). US · In-Market · Read report → DL · Rare Disease · CI Team · Medical Affairs US PNH Disease Landscape PNH diagnosis pathway, FLAER flow-cytometry bottleneck and the treated-prevalent pool across US centres. US · In-Market · Read report → P&HTA · Rare Disease · Market Access · Medical Affairs US PNH Payer & HTA Why the orphan-drug exclusion shields anti-C5 agents from IRA negotiation, ICER's 2024 value verdict on iptacopan, and Part B vs Part D routing. US · In-Market · Read report → DL · Rare Disease · CI Team · Launch Lead · Market Access US Myasthenia Gravis Disease Landscape Neuromuscular-junction autoantibody biology, the AChR/MuSK/seronegative split, and the crisis burden that defines US gMG. US · In-Market · Read report → CI · Rare Disease · CI Team · Launch Lead · Market Access US Myasthenia Gravis Competitive Intelligence Three novel mechanisms in 24 months — FcRn antagonists vs C5 inhibitors, and the AChR+ vs MuSK+ line that segments the market. US · In-Market · Read report → P&HTA · Rare Disease · CI Team · Launch Lead · Market Access US Myasthenia Gravis Payer & HTA ICER priced efgartigimod's value at $18,300–28,400 a year, under half its ~$418,400 launch cost. That gap is hardening into a three-tier FcRn-to-C5 step-edit across US commercial plans. US · In-Market · Read report →