The Committee for Medicinal Products for Human Use (CHMP) at the European Medicines Agency (EMA) gave a positive opinion, recommending the granting of a conditional marketing authorisation for Crizanlizumab (Adakveo®), intended for the prevention of recurrent vaso-occlusive crisis (VOCs) and subsequent pain crisis in patients with Sickle Cell Disease from 16 years of age.

Sickle Cell Disease is a complex disorder with very serious manifestations that impact on patients' quality of life. Although it is considered a rare disease, tens of thousands of people in Europe suffer from Sickle Cell Disease.

Crizanlizumab significantly reduces the rate of vaso-occlusive crisis, by binding to a cell adhesion protein that plays a central role in the vaso-occlusion process, and it can be administered in combination with Hydroxyurea or as monotherapy in patients for whom this drug is inadequate.

"Although Sickle Cell Disease has been recognised as a global public health issue affecting millions of people worldwide, including children, as it occurs early in the first years of life, the drug has been approved only over the age of 16. This means that, as in many other conditions, currently there are no completed paediatric studies, so Adakveo is in addition to many drugs authorised in Europe and around the world, excluding children," stresses Professor Adriana Ceci (Fondazione Benzi and TEDDY Board) who adds: " therefore, this drug, that is crucial for all SCD patients, at best will be authorized years too late and at worst will be used 'off-label' at all age ranges with all the risks that this use entails".

Currently, the Paediatric Investigation Plan (PIP) has been approved by the Paediatric Committee of the EMA. It aims at investigating the effects of Crizanlizumab on the prevention of vaso-occlusive crisis (VOC) in children with Sickle Cell Disease from 6 months to 18 years of age. Moreover, a study aimed to assess the drug appropriate dose and safety in paediatric patients from 6 months to 17 years of age with vaso-occlusive crisis is ongoing.

Keep following us for further updates and visit the TEDDY website to find out more information about the paediatric research!

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