Gate2Brain receives EMA Orphan Drug Designation for pioneering treatment of paediatric brain tumours

Gate2Brain, a spin-off from the Sant Joan de Déu Barcelona Children’s Hospital, IRB Barcelona and the University of Barcelona, has achieved a major milestone in paediatric cancer research. The European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its innovative therapeutic candidate, G2B-002, for the treatment of aggressive paediatric brain tumours such as diffuse intrinsic pontine glioma (DIPG) and paediatric glioblastoma (pGBM).

A breakthrough in paediatric oncology

The EMA’s Orphan Drug Designation recognises G2B-002’s potential to treat rare and severe diseases affecting fewer than 5 in 10,000 people in the EU, particularly those with limited or no existing treatment options. This recognition is a significant step towards changing the treatment landscape for childhood brain tumours.

G2B-002 utilises Gate2Brain’s proprietary peptide-based technology designed to improve drug delivery across the blood-brain barrier (BBB). By increasing precision and efficacy, this approach enhances the therapeutic potential of drugs targeting brain tumours while minimising side effects.

Focus on high impact paediatric diseases

Gate2Brain’s primary focus is on DIPG and pGBM – two of the most aggressive paediatric brain tumours with extremely poor prognoses. These cancers are among the leading causes of cancer-related mortality in children, highlighting the urgent need for innovative solutions.

“We are moving towards clinical trials that could transform the treatment of paediatric brain tumours,” said Meritxell Teixidó, CEO of Gate2Brain. She underlined the company’s strong commitment to paediatric patients and their families to address the critical unmet needs in this area.

Looking ahead

With the EMA ODD in hand, Gate2Brain is poised to accelerate the clinical development of G2B-002. Its success could pave the way for new therapies, not only for paediatric brain tumours, but potentially for other diseases requiring precision drug delivery across complex biological barriers.

An innovation hub for paediatric care

As a spin-off of the Sant Joan de Déu Barcelona Children’s Hospital, Gate2Brain exemplifies the mission of i4KIDS, the paediatric innovation hub coordinated by the hospital. By supporting cutting-edge technologies such as G2B-002, i4KIDS reinforces its commitment to improving paediatric care through innovation, collaboration and research, with a special focus on rare diseases, most of which manifest themselves during childhood.