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#1
Sat Feb 17, 2024, 01:04 PM
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FDA approves new drug for PNH
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Marlene, wife to John DX w/SAA April 2002, Stable partial remission; Treated with High Dose Cytoxan, Johns Hopkins, June 2002. Final phlebotomy 11/2016. As of July 2021 HGB 12.0, WBC 4.70/ANC 3.85, Plts 110K. 
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#2
Thu Jul 11, 2024, 06:20 PM
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The oral drug is iptacopan (trade name Fabhalta). It was found to improve hemoglobin levels and reduce the need for transfusions in adults with PNH.
Iptacopan is a complement factor B inhibitor, which means that it binds to factor B of the alternative complement pathway, preventing hemolysis. In trials it was found to be a better treatment than anti-C5 therapy. Side effects are mostly minor, such as cold symptoms, diarrhea, or rashes, but there's some risk of infections.
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Founder of Marrowforums and caregiver for my wife
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