Gate2Brain Gains Orphan Status

Gate2Brain, an IRB Barcelona spin-off, has secured the European Medicines Agency Orphan Drug Designation for G2B-002, a peptide-shuttle therapy that carries an anticancer agent across the blood–brain barrier to treat diffuse intrinsic pontine glioma and paediatric glioblastoma. The recognition provides fee reductions, protocol assistance and ten years of market exclusivity, thereby accelerating the move toward first-in-human trials.

Gate2Brain’s platform remains applicable to many drugs that currently fail to reach the brain, so the regulatory boost not only de-risks its lead asset but also validates the broader technology for CNS disorders. For children facing lethal tumours with no effective treatment options, the designation brings a potential breakthrough therapy several steps closer to clinical practice.

More information: Gate2Brain Receives EMA Orphan Drug Designation for Its Pediatric Cancer Treatment