Article
Source: Campus Sanofi
8 May 2026

Tolerability of Accelerated Agalsidase Beta Infusions in Fabry Disease: The SHORTEN Study Analysis

Author: Sanofi Editorial Team

Sanofi Editorial Team

Evaluating Safety and Tolerability of Differing Infusion
rates of Agalsidase beta for Fabry Disease - An
Analysis from the SHORTEN study

	Emerging evidence
• A Japanese post-marketing study showed patients <30 kg with classic FD tolerated 1 mg/kg/hour, completing infusions in 60 minutes.¹

	Unmet evidence gap
• Safety of <90-minute infusions is not well established.¹ • Limited data on infusion-associated reactions and adverse events at higher rates.¹

	Study objective
• To evaluate safe infusion-rate escalation to reduce the long-term treatment burden of agalsidase beta.^1,2

	Study design and eligibility criteria
The study was designed as a phase 4, open-label, single-arm, multicenter trial.^1,2

Patient population¹
Inclusion criteria	Exclusion criteria
• Participants aged ≥2 to ≤65 years with confirmed FD • Agalsidase beta–experienced patients enrolled in Cohorts 1–4 - Cohort 1: n=3 females, body weight ≥30 kg - Cohort 2: n=3 non-classic males, body weight ≥30 kg - Cohort 3: n=4 classic males, body weight ≥30 kg - Cohort 4: n=4 patients, body weight <30 kg - Cohort 5: Planned inclusion of ERT-naïve patients	• Pregnant or breastfeeding women • Known hypersensitivity or contraindication to agalsidase beta • Any medical condition considered to make increased infusion rates intolerable

Abbreviations

αGAL ERT: Alpha-galactosidase enzyme replacement therapy, FD: Fabry disease, mg/kg: milligrams per kilogram, FDA: Food and Drug Administration, lyso-GL3: globotriaosylsphingosine.

References

MAT-BH-2600200 - v1- April2026

Abbreviations

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