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Abstract: SA-PO655

A Phase 4 Study to Evaluate the Safety and Tolerability of Higher Infusion Rates of Agalsidase Beta to Shorten Infusion Duration in Fabry Disease: Interim Analysis

Session Information

Category: Genetic Diseases of the Kidneys

  • 1202 Genetic Diseases of the Kidneys: Non-Cystic

Authors

  • Banikazemi, Maryam, Department of Human Genetics, New York Medical College, New York, New York, United States
  • Goker-Alpan, Ozlem, Lysosomal Disorders Research and Treatment Unit, Fairfax, Virginia, United States
  • Nedd, Khan J., Infusion Associates, Grand Rapids, Michigan, United States
  • Maski, Manish, Sanofi, Cambridge, Massachusetts, United States
  • Lee, Chase, Sanofi, Cambridge, Massachusetts, United States
Background

Recombinant human α-galactosidase A (agalsidase beta), an enzyme replacement therapy (ERT) for treatment of Fabry disease (FD), is dosed biweekly at 1 mg/kg body weight, and infusion rates can be increased based on tolerability. The FDA label specifies ≥ 90 min infusions for all patients and maximum infusion rate of 15 mg/hr in patients ≤ 30 kg. The present study is evaluating the safety and tolerability of agalsidase beta at approved dose at an increased infusion rate with reduced total infusion volume.

Methods

This ongoing Phase 4 (NCT06019728), open label, single arm study will enroll 18 participants (≥2 to ≤65 years) with confirmed diagnosis of FD into five cohorts. The primary endpoints are percentage reduction of infusion duration from prestudy mean of the most recent three infusions and from an initial 120 minutes, along with shortest infusion duration each participant can tolerate. Here we present data from Cohort 1 and part of Cohort 2, with three heterozygous females and one male with variants in the GLA gene. All 4 patients were previously treated with agalsidase beta without any infusion associated reaction (≥ 30 kg).

Results

Three female patients aged 22, 19, and 49 years (mean body weight, 70.7 kg) and one male patient (age: 61 years, body weight: 100 kg) were enrolled. The duration of previous agalsidase beta treatment was 132, 60, 58, 43 months, and the prestudy mean duration of the three most recent infusions was 133, 136, 120, 90 minutes, respectively. The initial duration for first infusion in all three female patients was 120 minutes and was 60 minutes for the male patient. In subsequent infusions (four for the female patients and 2 for the male patient), the infusion time was gradually reduced to 20 minutes without any patient experiencing any infusion associated reaction (IAR). The plasma lyso-GL3 remained low and stable, and antidrug antibodies to agalsidase beta remained negative, in all four patients throughout the study.

Conclusion

Agalsidase beta infusion was given safely at a final time of 20-minute infusion duration in ERT-experienced female and male patients without any IAR. Further findings from this study will help in establishing a protocol to reduce duration of agalsidase beta infusion and minimize the treatment burden.

Funding

  • Commercial Support – Sanofi