Agalsidase beta biosimilar(ISU Abxis Co., Ltd.)

ISU303 is a new recombinant agalsidase beta (Agal) enzyme replacement therapy under investigation for Fabry disease, caused by a deficiency in α‐galactosidase A activity that leads to fatty deposits in tissues. We evaluated the pharmacokinetic (PK) parameters, safety and tolerability of ISU303 in healthy adult volunteers. The study was a dose block‐randomized, double‐blinded, placebo‐controlled, single‐dosing, and dose escalation phase 1 clinical trial. A total of 18 healthy subjects were enrolled (0.3 mg/kg, n = 6; 1.0 mg/kg, n = 6; placebo, n = 6). Blood samples for PK analysis were collected according to planned time. The PK parameters in each 0.3 and 1.0 mg/kg Agal group were as follows: Cmax (mU/mL) 43.19 ± 5.9 and 195.86 ± 32.3; AUClast (h·mU/mL) 207.91 ± 25.1 and 939.96 ± 158.3; t1/2 (hours) 1.13 ± 0.3 and 1.46 ± 0.2; Cl (mL/min/kg) 1.79 ± 0.2 and 1.34 ± 0.2, respectively. There were seven adverse events (AE) overall. All AEs were resolved without any complications. None were related to the study drug. There were no immunogenicity or any significant infusion‐related reactions. The new Agal product exhibited a dose‐dependent PK and was well tolerated with no significant AEs in healthy adult volunteers.