Gate2Brain receives orphan drug designation from the United States Food and Drug Administration
The biotechnological spin-off of the University of Barcelona, Gate2Brain, an innovator in drug delivery systems across biological barriers, has announced that its lead candidate, G2B-002, has received orphan drug designation (ODD) from the United States Food and Drug Administration (FDA). This grant is in addition to that already granted by the European Medicines Agency (EMA) at the end of 2024 and highlights the great potential of G2B-002 for the treatment of rare and aggressive brain tumors in pediatric and adult patients.
G2B-002 is designed to combat diffuse intrinsic pontine glioma and pediatric glioblastoma, two of the most lethal pediatric brain tumors with few therapeutic options. Through Gate2Brain’s patented peptide-based technology, G2B-002 optimizes the drug’s transport and penetration across the blood-brain barrier, increasing its efficacy and minimizing its systemic side effects.
With this FDA designation, G2B-002 will benefit from enhanced regulatory support and a strategic boost that will facilitate its transition from advanced preclinical to clinical trials.
“The orphan drug designation by the EMA and FDA reinforces our confidence in the potential of G2B-002 to meet critical needs in pediatric oncology,” says Meritxell Teixidó, CEO and CSO of Gate2Brain. “This double recognition brings us one step closer to clinical trials and, ultimately, to offering new hope to young patients and their families.”
Gate2Brain was founded in 2020 as a spin-off of IRB Barcelona, the University of Barcelona and the Hospital Pediàtric Sant Joan de Déu. Today, the company maintains its purpose of transforming the therapeutic landscape of pediatric brain tumors through innovative solutions in drug delivery.