Population Pharmacokinetic Modeling for the Iminosugar Lucerastat Supports Dose Adaptation in Patients With Fabry Disease and Moderate to Severe Renal Function Impairment.

What was studied

This study built a computer model of how lucerastat moves through the body. Lucerastat is a drug being studied for Fabry disease. The model used data from 250 people in early and late studies. It checked how weight, disease status and kidney function change drug levels. It then ran simulations.

What they found

Lucerastat clearance was lower in people with lower kidney filtering, called eGFR. Body weight and disease status had limited effect on drug levels. The model fit the data well across doses from 100 to 4000 mg. Only kidney problems called for dose changes. The dose steps used in the late trial gave similar drug levels. This held across kidney function groups.

What it means for patients

The authors say the model supports changing the dose in people with kidney impairment. In the late trial, doses ran from 1000 mg down to 250 mg. The dose was set by kidney filtering level.

Why it is not certain yet

This is a modeling study, not a test of whether the drug works. The abstract gives no results on health benefits. The model results rest on simulations. The abstract does not say how many people had severe kidney impairment.

Source

Population Pharmacokinetic Modeling for the Iminosugar Lucerastat Supports Dose Adaptation in Patients With Fabry Disease and Moderate to Severe Renal Function Impairment.

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