Gate2Brain obtains FDA Orphan Drug Designation for G2B-002
- US FDA grants Orphan Drug Designation for G2B-002 in 2025, underscoring U.S. regulatory support for this novel pediatric brain cancer therapy.
- EMA awarded Orphan Drug Designation in late 2024, marking dual-region recognition of G2B-002’s potential.
Gate2Brain, a Barcelona-based biotech innovator in drug delivery across biological barriers, announces 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 pediatric and adult brain tumors.
G2B-002 is designed to target diffuse intrinsic pontine glioma (DIPG) and pediatric 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.
“Receiving ODD from both the EMA and US FDA reaffirms our conviction in G2B-002’s potential to address critical unmet needs in pediatric oncology,” said Meritxell Teixidó, CEO & CSO of Gate2Brain. “This dual recognition brings us a step closer to clinical trials and, ultimately, to offering new hope for young patients and their families.”
Founded in 2020 as a spin-off from IRB Barcelona, University of Barcelona, and Sant Joan de Déu Pediatric Hospital, Gate2Brain remains dedicated to transforming the treatment landscape for pediatric brain tumors through innovative delivery solutions.
The company has received support from institutions including Fundación Botín (Mind the Gap), Banco Sabadell (BStartup Health), CDTI (Neotec, Multipaís), the European Innovation Council (EIC Accelerator), Startup Capital d’ACCIÓ and CaixaResearch Consolidate de la “Fundació la Caixa”.
Gate2Brain envisions improving patients’ quality of life through technology that enhances drug delivery efficiency.
