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

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