Selective BRAF Inhibitor Gets Orphan Drug Status for Primary CNS Tumors

The Food and Drug Administration (FDA) has granted Orphan Drug designation to FORE8394 for the treatment of primary brain and central nervous system (CNS) malignancies.

FORE8394 is a novel, small molecule, next-generation, orally available selective inhibitor of mutated BRAF. The investigational drug is intended to spare wild-type forms of RAF while targeting a wide range of BRAF mutations. According to FORE Biotherapeutics, FORE8394 is considered a “paradox breaker” as it does not induce paradoxical activation of the RAF/MEK/ERK pathway, and could therefore treat acquired resistance to current RAF inhibitors.

The Company is currently investigating the efficacy and safety of FORE8394 in the phase 3 FORTE trial (ClinicalTrials.gov Identifier: NCT05503797) in patients with advanced unresectable solid or primary CNS tumors harboring BRAF alterations. The trial will include adult and pediatric participants at least 10 years of age weighing at least 30kg.

“The receipt of Orphan Drug designation is another important regulatory achievement that reinforces the FDA’s recognition of the potential of FORE8394 to improve clinical outcomes in patients with BRAF-altered brain tumors,” said Stacie Shepherd, MD, PhD, Chief Medical Officer of Fore Biotherapeutics. “This designation will help us continue to expedite the development of our novel BRAF inhibitor, and we look forward to working closely with the global investigator community supporting FORTE and to advancing the development of FORE8394 for patients in need.”