Rare disease but nice to have an effective oral treatment option for PNH. Iptacopan, a first-in-class oral factor B inhibitor, has shown to improve Hb by >2g in the vast majority of patients in two studies discussed here.
Rare disease process that can carry significant morbidity. It is encouraging to see 40% response in such patient population when no other therapies (this is a first-in class) exist besides supportive care with very favorable toxicity profile.
FCS Hematology Oncology Review creates a platform for our physician network to observe the most recent articles and studies available in the oncology and hematology world. By sharing these articles we are building our wealth of knowledge of new observations and treatments as they come available.
