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Explore the long-term data of FABHALTA through 2 years

Data from the rollover extension program (REP) of adults with PNH from APPLY and APPOINT through 2 years1

The data are for observation only. No formal conclusions can be made.

Data of adult patients in REP from APPLY or APPOINT through 2 years.

*Data from the REP are summarized descriptively.2
Percentages at 2 years are based on the number of patients with Hb results at that time point.2

Additional end points

Additional data of adult patients in REP from APPLY or APPOINT through 2 years.

Mean LDH* remained <1.5 ULN through 2 years on FABHALTA2

  • In patients from APPLY: 33.00 U/L change from baseline (SD; 230.261); baseline: 273.84 (SD; 94.25)

  • In patients from APPOINT: -1399.18 U/L change from baseline (SD; 652.417); baseline: 1698.78 (SD; 683.33)

  • In patients from APPLY and APPOINT: -428.99 U/L change from baseline (SD; 789.05); baseline: 692.94 (SD; 752.20)

*Data from the REP are summarized descriptively.2
Excludes values within 30 days post-transfusion.2
§The number of transfusions does not include the period Days 1-14 after starting LNP.2

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Definitions
FACIT-Fatigue, Functional Assessment of Chronic Illness Therapy-Fatigue; Hb, hemoglobin; LDH, lactate dehydrogenase; PNH, paroxysmal nocturnal hemoglobinuria; RBC, red blood cell; SD, standard deviation; ULN, upper limit of normal.

References
1. Data on file. Study CLNP023C12001B Clinical Trial Protocol. Novartis Pharmaceuticals Corp; 2023.
2. Data on file. Study CLNP023C12001B supporting analyses using 2-year efficacy data for US Medical deck. Novartis Pharmaceuticals Corp; 2025.