FORE Biotherapeutics Receives Breakthrough Therapy Designation for Plixorafenib in CNS Tumours
Thursday, April 02, 2026
FORE Biotherapeutics has announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to plixorafenib for the treatment of adult patients with BRAF V600E-mutated high-grade glioma (HGG).
High-grade gliomas are aggressive brain tumours with limited treatment options and poor outcomes. Current therapies are often associated with significant side effects, highlighting the need for more effective and better tolerated treatments.
Plixorafenib is a novel BRAF inhibitor designed to selectively target BRAF alterations. It acts as a dimer breaker and has shown a differentiated profile in patients with primary CNS tumours, including glioblastoma. Data from the Phase 1/2a study, previously presented at scientific meetings, showed a 67 per cent overall response rate in a subgroup of patients with BRAF V600-mutated primary CNS tumours who had not received prior MAPK inhibitor treatment.
Plixorafenib has previously received Fast Track Designation for cancers with BRAF Class 1 and Class 2 alterations and Orphan Drug Designation for primary brain and CNS malignancies. These regulatory pathways, along with the BTD, are expected to support its continued development for patients with BRAF-altered CNS tumours.
BRAF-altered recurrent primary CNS tumours remain an area of high unmet need, as currently available treatments have limited effectiveness and tolerability. Plixorafenib is being developed to address these limitations through its novel mechanism of action and promising clinical profile.
The company noted that this may be the first time a targeted therapy has received this designation for HGG.
The designation is based on data from around 25 patients enrolled in a completed Phase 1/2a study and the ongoing Phase 2 FORTE basket trial. The trial is evaluating plixorafenib in patients with BRAF V600E-mutated central nervous system (CNS) tumours, including high-grade gliomas, low-grade gliomas, and other primary brain and spinal cord tumours in both adults and children. BTD is intended to accelerate the development and regulatory review of medicines for serious conditions where early data show meaningful improvement over existing treatments.
The BRAF V600E CNS cohort in the FORTE trial has met its pre-specified interim analysis criteria. The Independent Data Monitoring Committee supported continuation of the study based on observed responses and ongoing safety monitoring.
With BTD status, FORE Biotherapeutics will receive closer guidance from the FDA, including more frequent interactions and the possibility of priority and rolling review of its marketing application. This could help speed up the development process.
Source: fore.bio
