“The Innovative Medicines Initiative is making a difference to children living with paediatric diseases. Children represent 20% of the EU population and yet many of them live with significant unmet medical need. Less than half of all medicines used by children today...
Ivacaftor (Kalydeco, Vertex Pharmaceuticals) is already FDA approved to treat cystic fibrosis in patients aged 6 months and older. The new approval was based on data from ARRIVAL, a phase 3, open-label, safety cohort study of six children with cystic fibrosis aged 4...
In October 2020 the European Medicines Agency (EMA) expressed positive opinion on the adoption and extension of indication for a number of treatments addressed to the paediatric population. This is the case of Dupixent (dupilumab), for which on 15 October 2020, the...
The European Paediatric Formulation Initiative (EuPFI) works to improve the preparation of better and safer medicines for children. One area they are interested in is the “gadgets” or devices (e.g. measuring spoons, dosing cups, inhalers) that are used to help...
The first General Assembly of the EPTRI (European Paediatric Translational Research Infrastructure) Italian Node (EPTRI-IT) was held online on October 28th, 2020. The 3 hours meeting was attended by more than 60 representatives coming from the 24 Italian Institutions...
On 31 August 2020, the Enpr-EMA (European Network of Paediatric Research at the European Medicines Agency) working group on trial preparedness published recommendations on preparedness of medicines’ clinical trials in paediatrics. Enpr-EMA held a two-month public...
On 25 November 2020, the European Commission published the inception impact assessment on the revision of the Orphan and Paediatric Regulation. EU legislation on medicines for children and rare diseases was introduced in the early 2000’s, to address the lack of...
The TEDDY Chair, Annagrazia Altavilla, consultant and expert of the Council of Europe, is very pleased to announce the approval by the Committee on Bioethics of Council of Europe of a Concept note on “Participation of children in the decision-making process on matters...
On 31 August 2020, the Enpr-EMA (European Network of Paediatric Research at the European Medicines Agency) working group on trial preparedness published recommendations on preparedness of medicines’ clinical trials in paediatrics. Enpr-EMA held a two-month public...
EMA has recommended granting a marketing authorisation in the European Union for the gene therapy Libmeldy to treat metachromatic leukodystrophy (MLD), a rare inherited metabolic disease that affects the nervous system. Libmeldy is indicated for use in children with...