The European Medicines Agency (EMA) has published the Human medicines highlights of 2022.

In 2022, EMA recommended 89 medicines for marketing authorisation. Most of them covers the oncology area followed by neurologic and endocrinologic diseases. Six are Advanced Therapy Medicinal Products and 21 were designated as orphan medical products.

The authorisation of new medicines is essential to advancing public health. Children should not be excluded from this relevant opportunity!

Looking at the paediatic population, among the medicines approved in 2022 representing significant progress in their therapeutic areas, the EMA report includes:

  • Xenpozyme, the first therapy for the treatment of adult and paediatric patients with acid sphingomyelinase deficiency (historically known as Niemann-Pick disease type A, A/B and B), a rare genetic condition
  • Zokinvy, the first treatment for children with progeroid syndromes, an ultrarare genetic disease which causes premature aging and death
  • Beyfortus, the first medicine for the prevention of respiratory syncytial virus lower respiratory tract disease in newborns and infants
  • Upstaza, the first treatment for adults and children with aromatic L amino acid decarboxylase deficiency, an ultra-rare genetic disorder affecting the nervous system
  • Ebvallo, for the treatment of adults and children with Epstein-Barr virus positive post-transplant lymphoproliferative disease

In addition, 90 extensions of indication were recommended in 2022, including 37 for paediatric use. The extension of the use of a medicine that is already authorised can offer new treatment opportunities for patients, including children.

Five medicines were authorised under exceptional circumstances. All of them are intended for children.  This is a way allowing patients access to medicines that cannot be approved under a standard authorisation as comprehensive data cannot be obtained, as often occurs for paediatric diseases.

The enhanced development support provided by the PRIME scheme aims at helping patients to benefit as early as possible from promising medicines that target an unmet medical need, by optimising the generation of robust data and enabling accelerated assessment. This year, 8 PRIME-designated medicines were recommended for approval, including 3 addressing paediatric diseases.

Advancements were achieved for children, but it is still a long way! A lot of efforts should still be done by all involved stakeholders including sponsors, academia, regulators and patients’ representatives.

Discover more by consulting the full EMA report here!

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