AXLRx Alexion (AZ Rare) intelligence — Rare Disease: 6 reports covering Rare Disease.
Iptacopan oral pivot versus the anti-C5 IV class. Orphan-drug exclusion from IRA negotiation (US), NICE HST (UK) and SFDA lag (GCC).
PNH diagnosis pathway, FLAER flow-cytometry bottleneck and the treated-prevalent pool across US centres.
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.
Three novel mechanisms in 24 months — FcRn antagonists vs C5 inhibitors, and the AChR+ vs MuSK+ line that segments the market.
ICER priced efgartigimod's value at $18,300 to $28,400 a year, under half its ~$418,400 launch cost, and that gap is hardening into a three-tier FcRn-to-C5 step-edit across US commercial plans.
Neuromuscular-junction autoantibody biology, the AChR/MuSK/seronegative split, and the crisis burden that defines US gMG.