# 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

## TL;DR

This study compares a new drug, Abcertin, to an existing treatment, Cerezyme, for Gaucher disease and finds they are similar in safety and effectiveness.

## Contribution

The study demonstrates pharmacokinetic bioequivalence and comparable safety of Abcertin to Cerezyme in healthy volunteers.

## Key findings

- Abcertin showed pharmacokinetic equivalence to Cerezyme with similar peak concentrations and area under the curve.
- Abcertin had a comparable safety and tolerability profile with no clinically significant adverse events.
- Only one participant developed non-neutralizing anti-drug antibodies, indicating low immunogenicity.

## Abstract

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.

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.

Abcertin reached peak plasma concentrations at a median t
max of 61 min (range: 40–121 min). The mean C
max, 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 t
½, CL, and V
z 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.

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.

This study evaluates the pharmacokinetic equivalence, immunogenicity, and safety profiles of Abcertin and Cerezyme in healthy volunteers. It demonstrated the pharmacokinetic bioequivalence of Abcertin to Cerezyme, highlighted favorable safety and tolerability profiles with no clinically significant adverse events for Abcertin, and confirmed its low immunogenic potential.