AXLRx AstraZeneca intelligence — Rare Disease in United States: 2 reports covering Rare Disease in United States.
Tagrisso dominant in first-line EGFR+ NSCLC; Alexion complement franchise (Ultomiris/Soliris) holds strong specialty access.
Iptacopan oral pivot versus the anti-C5 IV class. Orphan-drug exclusion from IRA negotiation (US), NICE HST (UK) and SFDA lag (GCC).
Three novel mechanisms in 24 months — FcRn antagonists vs C5 inhibitors, and the AChR+ vs MuSK+ line that segments the market.