AXLRx BioCryst intelligence — Rare Disease in United States: 3 reports covering Rare Disease in United States.
The prophylaxis class is fracturing along route: oral berotralstat and the first oral on-demand agent against a still-injectable antibody field.
HAE pathophysiology, the Type I/II split, attack burden and the diagnostic-delay problem that defines the US in-market landscape.
Specialty-tier prior authorization, prophylaxis above $300K/patient/yr, ICER 2018/2021 value-based benchmarks and no-concurrent-acute-agent rules.