General Court upholds European Medicines Agency decision to refuse paediatric waiver for perflubutane

News 15/12/2011

The General Court of the European Union has issued a judgement stating that the European Medicines Agency acted appropriately when it rejected the application for a paediatric waiver for the diagnostic agent perflubutane.

The judgement, issued yesterday following a hearing in Luxembourg on 12 July 2011, dismissed the application made by Nycomed Danmark ApS concerning the Agency's decision to reject its application to waive the requirements to investigate the use of perflubutane in children.

The company applied for the waiver in 2008, stating that its intended indication for the medicine was in the diagnosis of coronary artery disease, which only occurs in adults.

The Agency rejected this application on the grounds that the intended use of the medicine was in fact to improve the visibility of blood flow in the heart muscle during an ultrasound scan. This can be used to diagnose a range of underlying diseases, including some diseases that occur in children. The Agency therefore concluded that a waiver was not appropriate.

In upholding the Agency's decision, the General Court supported the interpretation of the Paediatric Regulation as understood by the Agency, agreeing that its Paediatric Committee (PDCO) expressed its opinion on objective, scientifically driven arguments. It confirmed that indications applied for by companies should only be considered as the starting point for the PDCO's assessment and that the Committee should take the properties of a medicine into account when considering its potential uses in the treatment and care of children.

The Court also rejected the company's claim that the Agency misused its powers.

The Paediatric Regulation aims to make medicines available for children, which are fully developed and assessed for use in ethical clinical trials. It states that all applications for marketing authorisation for new medicines have to include the results of studies carried out in children of different ages. The PDCO determines what these studies must be and describes these in paediatric investigation plans (PIPs).

Pharmaceutical companies can have this obligation waived if they can provide evidence that the conditions that the medicine can be used for only occur in adults.

During the case, the Agency was supported by the European Commission, Portugal, Belgium, the United Kingdom and France. The company has the right to appeal against the judgement.

How useful was this page?

Add your rating