Pegunigalsidase Alfa Elfabrio® as a Long-Term Enzyme Replacement Therapy in Adults With Fabry Disease: A Systematic Literature Review
Author(s)
Geetank Kamboj, MPharm1, Shainki Sharma, MPharm1, Surabhi Aggarwal, MPharm1, George Papadopoulos, BSc (Hons)2, Steven Crowley, MSc, MBA2, Hemant Rathi, MSc1.
1Skyward Analytics, Gurugram, India, 2Lucid Health Consulting, Sydney, Australia.
1Skyward Analytics, Gurugram, India, 2Lucid Health Consulting, Sydney, Australia.
OBJECTIVES: To evaluate the efficacy and safety of pegunigalsidase alfa (Elfabrio®) compared to the other enzyme replacement therapies (ERTs) in adults with Fabry disease through a systematic literature review.
METHODS: A structured literature search was conducted to identify randomised controlled trials (RCTs) and single-arm studies from PubMed, Cochrane Library, US and WHO clinical trials registries up to January 08, 2025. Title-abstract and full-text screening were performed using the EasySLR™ platform. Studies that evaluated the efficacy and safety of pegunigalsidase alfa in comparison with agalsidase alfa and beta, or as monotherapy were included. Studies assessing only agalsidase alfa or beta were excluded.
RESULTS: A total of 41 publications covering seven studies were included in the final review. These included one randomized controlled trial, BALANCE (NCT02795676), which compared pegunigalsidase alfa with agalsidase beta, and six supportive non-randomized or open-label trials assessing pegunigalsidase alfa: BRIDGE (NCT03018730), BRIGHT (NCT03180840), BRIGHT-51 (NCT03614234), PB-102-F01 (NCT01678898), PB-102-F02 (NCT01678898), and PB-102-F03 (NCT01981720). No head-to-head trial comparing pegunigalsidase alfa with agalsidase alfa was identified. The BALANCE trial demonstrated non-inferior efficacy of pegunigalsidase alfa compared to agalsidase beta in ERT-experienced patients. Supportive studies provided additional evidence in both switch and treatment-naïve populations, including long-term safety and sustained efficacy. Overall, the included evidence supports comparable efficacy and a favorable safety and immunogenicity profile for pegunigalsidase alfa relative to existing ERTs. Although no head-to-head trial comparing pegunigalsidase alfa with agalsidase alfa was identified, an international cohort study by Arends et al., 2018 reported no significant differences in clinical outcomes between agalsidase alfa and beta.
CONCLUSIONS: Pegunigalsidase alfa demonstrated improved efficacy and a comparable safety profile to agalsidase beta, supporting its long-term use in adults with Fabry disease but further research is warranted to assess its comparative effectiveness versus agalsidase alfa.
METHODS: A structured literature search was conducted to identify randomised controlled trials (RCTs) and single-arm studies from PubMed, Cochrane Library, US and WHO clinical trials registries up to January 08, 2025. Title-abstract and full-text screening were performed using the EasySLR™ platform. Studies that evaluated the efficacy and safety of pegunigalsidase alfa in comparison with agalsidase alfa and beta, or as monotherapy were included. Studies assessing only agalsidase alfa or beta were excluded.
RESULTS: A total of 41 publications covering seven studies were included in the final review. These included one randomized controlled trial, BALANCE (NCT02795676), which compared pegunigalsidase alfa with agalsidase beta, and six supportive non-randomized or open-label trials assessing pegunigalsidase alfa: BRIDGE (NCT03018730), BRIGHT (NCT03180840), BRIGHT-51 (NCT03614234), PB-102-F01 (NCT01678898), PB-102-F02 (NCT01678898), and PB-102-F03 (NCT01981720). No head-to-head trial comparing pegunigalsidase alfa with agalsidase alfa was identified. The BALANCE trial demonstrated non-inferior efficacy of pegunigalsidase alfa compared to agalsidase beta in ERT-experienced patients. Supportive studies provided additional evidence in both switch and treatment-naïve populations, including long-term safety and sustained efficacy. Overall, the included evidence supports comparable efficacy and a favorable safety and immunogenicity profile for pegunigalsidase alfa relative to existing ERTs. Although no head-to-head trial comparing pegunigalsidase alfa with agalsidase alfa was identified, an international cohort study by Arends et al., 2018 reported no significant differences in clinical outcomes between agalsidase alfa and beta.
CONCLUSIONS: Pegunigalsidase alfa demonstrated improved efficacy and a comparable safety profile to agalsidase beta, supporting its long-term use in adults with Fabry disease but further research is warranted to assess its comparative effectiveness versus agalsidase alfa.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO178
Topic
Clinical Outcomes, Study Approaches
Topic Subcategory
Clinical Outcomes Assessment, Comparative Effectiveness or Efficacy
Disease
Biologics & Biosimilars, Rare & Orphan Diseases