Objective
Children are often treated as small adults—not as the special patients they are. Regulations exist to encourage the development of drugs and formulation for children. However, off-patent drugs are often administered to children without appropriate formulations or systematic investigations. Therefore, the paediatric use marketing authorisation (PUMA) has been designed.
The EMA Paediatric Working Party has prioritized the unmet need for the cardiovascular off-patent drug enalapril in the European paediatric population. Enalapril is an ACE inhibitor commonly used for cardiac failure in children under 1 year of age, although it is not labelled for any condition in patients <20 kg in European countries.
Current problems with the use of enalapril in children include: no appropriate formulation and insufficient stability of the liquid soluble formulation for young paediatric patients; insufficient paediatric pharmacokinetic (PK) data for any formulation; insufficient safety data in neonates, infants, and young children; and limited pharmacodynamics (PD) and efficacy data for paediatric cardiological indications.
The aim of the LENA (Labeling of Enalapril from Neonates up to Adolescents) project is to address these shortcomings and provide a basis for a future PUMA of enalapril by developing an age-appropriate solid oral formulation suitable for all paediatric subsets; generating PK and PD data; collecting data on the safety of enalapril in young paediatric patients; and providing dose recommendations based on PK/PD modelling and bridging from adult data.
The international collaboration of the LENA investigators will provide the required expertise to develop and evaluate drug formulation, investigate dosing, and recruit paediatric patients for clinical trials while respecting current legislation, regulatory considerations, and ethical aspects, with the aim of applying for a PUMA.
Call for proposal
FP7-HEALTH-2013-INNOVATION-1
See other projects for this call
Funding Scheme
CP-FP - Small or medium-scale focused research projectCoordinator
40225 Dusseldorf
Germany
See on map
Participants (13)
45721 HALTERN AM SEE
See on map
1970 WEZEMBEEK OPPEM
See on map
1096 BUDAPEST
See on map
3015 GD Rotterdam
See on map
11000 BEOGRAD
See on map
5020 Salzburg
See on map
EC2A 3NW LONDON
See on map
Participation ended
80333 Muenchen
See on map
WC1N 3JH London
See on map
3000 Leuven
See on map
1090 Wien
See on map
3584 CX Utrecht
See on map
75008 Paris
See on map