Multi-Year Registry Study of Elapegademase Treatment in Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Requiring Enzyme Replacement Therapy

• DOI: 10.1007/s10875-025-01873-3
• Publication Date: 2025-03-30T03:22:39Z
• Authors: Morna J. Dorsey; Manish J. Butte; Jay A. Lieberman; Heather Lehman; Tracy Fausnight; Michael D. Keller; Caroline Fradette; Michael S. Hershf...; Tamara C. Pozos; Anna Rozova; Luke A. Wall; Jeffrey J. Bednarski; Teresa K. Tarrant; Hey J. Chong; Bob Geng; Noemi Toiber Temin; Susan S. Laubach; Leo Lin; Talal Mousallem; Jolan E. Walter
• Keywords: Adenosine deaminase deficiency; Medical microbiology

Abstract Purpose The safety and tolerability of elapegademase (elapegademase-lvlr; Revcovi ® ) a PEGylated recombinant adenosine deaminase (ADA), were demonstrated in two Phase 3 clinical trials the U.S. Japan patients with ADA-deficient severe combined immunodeficiency (ADA-SCID). Elapegademase replaced Adagen (pegademase, bovine ADA) 2018. This registry study (NCT03878069) was conducted as post-marketing requirement to bolster limited effectiveness data on ADA-SCID starting enzyme replacement therapy (ERT) de novo . Methods Patients managed by routine care treating physicians’ judgement from September 2019 January 2023. Primary endpoints included trough plasma ADA activity total erythrocyte deoxyadenosine nucleotides (dAXP). Secondary outcomes lymphocyte counts, hospitalizations, infections, outcomes. Results Thirty-two grouped ERT-naïve ( n = 7; infants children no prior ERT [EN]); pegademase-transitioning 21; pegademase [PT]); who had participated trial 4; STP-2279-002; [STP]). EN group maintained optimal activity, increased manageable mortality for up 30 months while elapegademase. STP 66.7% PT continued maintain satisfactory levels both dAXP stable rates infections hospitalizations counts 48.6 months. Variability all measures seen, but overall, did not deteriorate Conclusion Effectiveness 4 years use new concerns.