EMA and FDA propose new approach for Gaucher disease drug development


The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) have proposed new approaches to the development of medicine to treat Gaucher disease.

Gaucher disease is a genetic disorder in which glucocerebroside, a sphingolipid also known as glucosylceramide, accumulates in cells and organs.

The new approaches will help facilitate the discovery of medicines that can be used for rare paediatric diseases in general.

The EMA and FDA's proposal discusses possible ways to improve the efficiency of medicine development for Gaucher disease, which is used as a model to evaluate recent progress made in the area of data extrapolation.

It enables medicine developers to extrapolate available clinical data, through appropriate modelling and simulation techniques, to determine the effectiveness of a medicine in children and adolescents.

"It enables medicine developers to extrapolate available clinical data to determine the effectiveness of a medicine in children and adolescents."

It will also allow developers to test the safety and efficacy of medicines manufactured by different companies in a single trial.

As the same control arm is used to compare more than one medicine under evaluation, the new approach supports the clinical testing of medicines, while reducing the total number of paediatric patients included in trials.

The joint proposal focuses on reducing the number of patients required for conducting clinical trials.

In addition, the EMA is finalising a reflection paper which highlights a systematic approach to scientifically reliable extrapolation of data to support medicine authorisation.


Image: Micrograph showing crinkled paper macrophages in the marrow space in a case of Gaucher disease. Photo: courtesy of Nephron / Wikipedia.