A Randomized, Double‐Blind, 2‐Treatment, 2‐Period, Crossover Phase 1 Study to Compare the Pharmacokinetics, Safety and Tolerability of 60 IU/Kg of Abcertin and Cerezyme in Healthy Volunteers Following a Single Intravenous Administration
ABSTRACT Background Imiglucerase (Cerezyme; Sanofi, Paris, France), an analogue of β‐glucocerebrosidase produced by recombinant DNA technology, has been a safe and effective treatment for Gaucher disease (GD) for over 25 years. A new imiglucerase, Abcertin (Seongnam‐si, Gyeonggi‐do, Republic of Kore...
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2025-06-01
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| Online Access: | https://doi.org/10.1002/mgg3.70111 |
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| author | Eungu Kang Dohyung Kim Soojin Hwang Charlotte Lemech Jessica Wharton Yongyoon Lee Han Wook Yoo Beom Hee Lee |
| author_facet | Eungu Kang Dohyung Kim Soojin Hwang Charlotte Lemech Jessica Wharton Yongyoon Lee Han Wook Yoo Beom Hee Lee |
| author_sort | Eungu Kang |
| collection | DOAJ |
| description | ABSTRACT Background Imiglucerase (Cerezyme; Sanofi, Paris, France), an analogue of β‐glucocerebrosidase produced by recombinant DNA technology, has been a safe and effective treatment for Gaucher disease (GD) for over 25 years. A new imiglucerase, Abcertin (Seongnam‐si, Gyeonggi‐do, Republic of Korea) has shown a similar safety and efficacy profile in previous clinical studies. This study compared the pharmacokinetics, immunogenicity, safety, and tolerability to EU‐sourced Cerezyme following a single 60 IU/kg dose. Methods This phase 1, single‐center, randomized, double‐blind, two‐way crossover study enrolled 36 healthy volunteers aged 18–45 years. Participants were randomly assigned to receive either Abcertin or Cerezyme in a predetermined sequence. Results Abcertin reached peak plasma concentrations at a median tmax of 61 min (range: 40–121 min). The mean Cmax, AUC0–last, and AUC0–inf were 115.4 mU/mL, 12,190 min·mU/mL, and 12,210 min mU/mL, respectively, indicating bioequivalence to Cerezyme. The mean t½, CL, and Vz were 6.88 min, 376.7 mL/min, and 3.62 L, respectively, and were comparable between the two treatments. One participant in the Cerezyme group developed anti‐drug antibodies, which were non‐neutralizing A total of 24 subjects experienced treatment‐emergent adverse event (TEAE). The most common TEAE was headache (3 in the Abcertin group and 5 in the Cerezyme group), followed by general disorders and administration site condition (3 in Abcertin group and 5 in Cerezyme group). Two participants in the Cerezyme sequence experienced severe TEAEs: one had a urinary tract infection, and the other developed urticaria, which leading to study withdrawal. Conclusion Abcertin demonstrated pharmacokinetic equivalence to Cerezyme, with a comparable safety, immunogenicity, and tolerability profile. These findings support its potential as an affordable biosimilar for GD treatment. |
| format | Article |
| id | doaj-art-dfe70de07c0645fca3b6dbe0903768a2 |
| institution | Kabale University |
| issn | 2324-9269 |
| language | English |
| publishDate | 2025-06-01 |
| publisher | Wiley |
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| series | Molecular Genetics & Genomic Medicine |
| spelling | doaj-art-dfe70de07c0645fca3b6dbe0903768a22025-08-20T03:26:29ZengWileyMolecular Genetics & Genomic Medicine2324-92692025-06-01136n/an/a10.1002/mgg3.70111A Randomized, Double‐Blind, 2‐Treatment, 2‐Period, Crossover Phase 1 Study to Compare the Pharmacokinetics, Safety and Tolerability of 60 IU/Kg of Abcertin and Cerezyme in Healthy Volunteers Following a Single Intravenous AdministrationEungu Kang0Dohyung Kim1Soojin Hwang2Charlotte Lemech3Jessica Wharton4Yongyoon Lee5Han Wook Yoo6Beom Hee Lee7Department of Pediatrics Korea University Ansan Hospital, Korea University College of Medicine Ansan Republic of KoreaDepartment of Pediatrics Asan Medical Center Children's Hospital, University of Ulsan College of Medicine Seoul Republic of KoreaDepartment of Pediatrics Asan Medical Center Children's Hospital, University of Ulsan College of Medicine Seoul Republic of KoreaScientia Clinical Research (SCR) Randwick New South Wales AustraliaScientia Clinical Research (SCR) Randwick New South Wales AustraliaISU ABXIS Co., Ltd. Seongnam‐si Gyeonggi‐do Republic of KoreaDepartment of Pediatrics CHA University School of Medicine, Bundang CHA Medical Center Seongnam Republic of KoreaDepartment of Pediatrics Asan Medical Center Children's Hospital, University of Ulsan College of Medicine Seoul Republic of KoreaABSTRACT Background Imiglucerase (Cerezyme; Sanofi, Paris, France), an analogue of β‐glucocerebrosidase produced by recombinant DNA technology, has been a safe and effective treatment for Gaucher disease (GD) for over 25 years. A new imiglucerase, Abcertin (Seongnam‐si, Gyeonggi‐do, Republic of Korea) has shown a similar safety and efficacy profile in previous clinical studies. This study compared the pharmacokinetics, immunogenicity, safety, and tolerability to EU‐sourced Cerezyme following a single 60 IU/kg dose. Methods This phase 1, single‐center, randomized, double‐blind, two‐way crossover study enrolled 36 healthy volunteers aged 18–45 years. Participants were randomly assigned to receive either Abcertin or Cerezyme in a predetermined sequence. Results Abcertin reached peak plasma concentrations at a median tmax of 61 min (range: 40–121 min). The mean Cmax, AUC0–last, and AUC0–inf were 115.4 mU/mL, 12,190 min·mU/mL, and 12,210 min mU/mL, respectively, indicating bioequivalence to Cerezyme. The mean t½, CL, and Vz were 6.88 min, 376.7 mL/min, and 3.62 L, respectively, and were comparable between the two treatments. One participant in the Cerezyme group developed anti‐drug antibodies, which were non‐neutralizing A total of 24 subjects experienced treatment‐emergent adverse event (TEAE). The most common TEAE was headache (3 in the Abcertin group and 5 in the Cerezyme group), followed by general disorders and administration site condition (3 in Abcertin group and 5 in Cerezyme group). Two participants in the Cerezyme sequence experienced severe TEAEs: one had a urinary tract infection, and the other developed urticaria, which leading to study withdrawal. Conclusion Abcertin demonstrated pharmacokinetic equivalence to Cerezyme, with a comparable safety, immunogenicity, and tolerability profile. These findings support its potential as an affordable biosimilar for GD treatment.https://doi.org/10.1002/mgg3.70111biosimilarenzyme replacement therapyGaucher diseasepharmacokineticssafety |
| spellingShingle | Eungu Kang Dohyung Kim Soojin Hwang Charlotte Lemech Jessica Wharton Yongyoon Lee Han Wook Yoo Beom Hee Lee A Randomized, Double‐Blind, 2‐Treatment, 2‐Period, Crossover Phase 1 Study to Compare the Pharmacokinetics, Safety and Tolerability of 60 IU/Kg of Abcertin and Cerezyme in Healthy Volunteers Following a Single Intravenous Administration Molecular Genetics & Genomic Medicine biosimilar enzyme replacement therapy Gaucher disease pharmacokinetics safety |
| title | A Randomized, Double‐Blind, 2‐Treatment, 2‐Period, Crossover Phase 1 Study to Compare the Pharmacokinetics, Safety and Tolerability of 60 IU/Kg of Abcertin and Cerezyme in Healthy Volunteers Following a Single Intravenous Administration |
| title_full | A Randomized, Double‐Blind, 2‐Treatment, 2‐Period, Crossover Phase 1 Study to Compare the Pharmacokinetics, Safety and Tolerability of 60 IU/Kg of Abcertin and Cerezyme in Healthy Volunteers Following a Single Intravenous Administration |
| title_fullStr | A Randomized, Double‐Blind, 2‐Treatment, 2‐Period, Crossover Phase 1 Study to Compare the Pharmacokinetics, Safety and Tolerability of 60 IU/Kg of Abcertin and Cerezyme in Healthy Volunteers Following a Single Intravenous Administration |
| title_full_unstemmed | A Randomized, Double‐Blind, 2‐Treatment, 2‐Period, Crossover Phase 1 Study to Compare the Pharmacokinetics, Safety and Tolerability of 60 IU/Kg of Abcertin and Cerezyme in Healthy Volunteers Following a Single Intravenous Administration |
| title_short | A Randomized, Double‐Blind, 2‐Treatment, 2‐Period, Crossover Phase 1 Study to Compare the Pharmacokinetics, Safety and Tolerability of 60 IU/Kg of Abcertin and Cerezyme in Healthy Volunteers Following a Single Intravenous Administration |
| title_sort | randomized double blind 2 treatment 2 period crossover phase 1 study to compare the pharmacokinetics safety and tolerability of 60 iu kg of abcertin and cerezyme in healthy volunteers following a single intravenous administration |
| topic | biosimilar enzyme replacement therapy Gaucher disease pharmacokinetics safety |
| url | https://doi.org/10.1002/mgg3.70111 |
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