*An acute TTP event was defined as a decrease in the platelet count by at least 50% from baseline or to less than 100,000 per μL and an elevation of the lactate dehydrogenase (LDH) level to more than 2 times the baseline value or more than 2 times the upper limit of the normal range (ULN).^2,3

† Subacute events were two or more of the following (including ≥1 laboratory measurement): a decrease in the platelet count by at least 25% from baseline or to less than 150,000 per μL, an increase in the lactate dehydrogenase (LDH) level to more than 1.5 times the baseline value or more than 1.5 times the upper limit of the normal range (ULN), or organ-specific signs or symptoms of TTP.^2,3

Adapted from Blood, Vol 142, Patel M, et al. Population Pharmacokinetics and Exposure-Response Analyzes to Demonstrate the Link of Causality between ADAMTS13 and the Clinical Effects of Recombinant ADAMTS13 Compared to Plasma-Based Therapies, Page 1260, Copyright 2023, with permission from ASH.

^{*Exposure-response model simulations suggested that >95% of patients treated with ADZYNMA are predicted to have plasma C_ave ADAMTS13 >0.13 IU/mL (equivalent to 13% activity), corresponding to a high probability (>70%) of protection against thrombocytopenia.⁴}

* Thrombocytopenia was defined as a decrease in the platelet count by ≥25% from baseline or to <150,000 per microlitre
† An elevated lactate dehydrogenase (LDH) level was defined as an increase to more than 1.5x the baseline value or more than 1.5x the upper limit of the normal range
‡ Increase in serum creatinine >1.5x baseline
§ The number of composite cTTP manifestations, an exploratory outcome measure, was defined as the occurrence of one or more of the following pre-specified isolated TTP manifestations: thrombocytopenia, elevated LDH level, increased creatinine level, neurologic symptoms or abdominal pain, excluding the "Others" category
¶ The ad hoc analysis of other TTP manifestations was intended to capture all TTP-related events reported by investigators that were not pre-specified TTP manifestations (e.g. fatigue or nausea)
** The widths of the confidence intervals have not been adjusted for multiplicity and should not be used to infer definitive treatment effects

Adapted from Scully M, et al. N Engl J Med. 2024;390(17):1584-1596.

Adapted from Scully M, et al. N Engl J Med. 2024;390(17):1584-1596.

*The pharmacokinetic (PK) profile of ADZYNMA was determined based on clinical trial ADAMTS13 activity data analyses. Following single-dose IV administration of ADZYNMA at 5 IU/kg, 20 IU/kg, and 40 IU/kg to adults and adolescents, dose-related increases in individual ADAMTS13 activity were observed and reached a maximum at approximately 1 hour post-administration or earlier.¹

1. ADZYNMA (rADAMTS13) EU Summary of Product Characteristics. Last approved version.
2. Scully M, et al. N Engl J Med. 2024;390(17):1584-1596. Supplementary appendix.
3. Scully M, et al. N Engl J Med. 2024;390(17):1584-1596.
4. Patel M, et al. Blood. 2023;142(Suppl 1):1260.
5. A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP): NCT03393975. Updated 4th June 2024. Accessed February 2025. Available at: https://clinicaltrials.gov/study/NCT03393975

ADAMTS13, A disintegrin and metalloproteinase with thrombospondin motifs 13
AUC, Area under the plasma concentration-time curve
C_ave, Average plasma concentration at steady-state
CI, Confidence interval
C_max, Maximum plasma concentration
cTTP, Congenital thrombotic thrombocytopenic purpura
CHO, Chinese hamster ovary
DNA, Deoxyribonucleic acid
ERT, Enzyme replacement therapy
FFP, Fresh frozen plasma
FVIII, Factor VIII
IU, International unit
IV, Intravenous
LDH, Lactate dehydrogenase
LLoQ, Lower limit of quantification
PBT, Plasma-based therapy
PK, Pharmacokinetic
Q1W, Once weekly
Q2W, Once every two weeks 
rADAMTS13, Recombinant ADAMTS13
S/D, Solvent detergent
SmPC, Summary of product characteristics
TTP, Thrombotic thrombocytopenic purpura
ULN, Upper limit of normal
vs, Versus

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