Rare Disease
We are committed to transforming care for patients with rare, complement-driven diseases.
There are over 7,000 rare diseases, of which only 5% have an approved treatment.1 We aim to help change this reality for patients and communities affected by these serious conditions.
In 2021, we brought forward the first new class of complement medicine in nearly 15 years with the approval of a treatment for PNH, a rare and potentially life-threatening blood disease.
In 2025, we advanced another first: the first approved treatment for patients 12 and older with C3G or primary IC-MPGN, rare and severe kidney diseases.
We strive to understand the unique challenges people living with a rare disease face and are committed to always putting patients first. This includes embedding the perspectives and needs of patients and their communities into our business decisions from research to clinical development and commercialization, and ensuring those who need our medicine have access as quickly as possible.
Advancing therapies across several rare diseases
Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is an acquired, rare, chronic, and potentially life-threatening blood disease that can appear at any age and in any race or gender, and is most often diagnosed in people in their early 30s.
Learn more » Learn more about our ongoing clinical trials »
C3 Glomerulopathy (C3G) and Primary Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
C3G and primary IC-MPGN are rare, severe kidney diseases that cause inflammation and damage to the organ. Without treatment, these diseases often lead to kidney failure within five to 10 years of diagnosis.2-4
Learn more »Resources
- NIH’s National Center for Advancing Translational Sciences
- Smith RJH, et al. Nat Rev Nephrol. 2019;15(3):129-143.
- Servais A, et al. Kidney Int. 2012;82(4):454-464.
- Zand L, et al. J Am Soc Nephrol. 2014;25(5):1110-1117.