The biotechnology company Gate2Brain, a member of CATALONIA.HEALTH, has announced that its lead candidate, G2B-002, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). This follows the European Medicines Agency’s ODD approval in late 2024 and highlights G2B-002’s potential in treating rare and aggressive paediatric and adult brain tumours.
G2B-002 is designed to target diffuse intrinsic pontine glioma (DIPG) and paediatric glioblastoma (pGBM), two of the most lethal childhood brain cancers with very limited treatment options. By leveraging Gate2Brain’s proprietary peptide-based delivery technology, G2B-002 enhances drug penetration across the blood–brain barrier, aiming to improve efficacy while reducing systemic side effects.
Endowed with this FDA designation, G2B-002 benefits from enhanced regulatory support and strategic momentum, smoothing its transition from advanced preclinical development into clinical trial phase.
Meritxell Teixidó, CEO & CSO of Gate2Brain, explained: “Receiving ODD from both the EMA and US FDA reaffirms our conviction in G2B-002’s potential to address critical unmet needs in paediatric oncology. This dual recognition brings us a step closer to clinical trials and, ultimately, to offering new hope for young patients and their families.”
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