Sino Biopharmaceutical drug TQ-B3234 gets breakthrough therapy designation

Sino Biopharmaceutical Limited announced today that its national Class 1 new drug, TQ-B3234, a selective MEK1/2 inhibitor, has been included in the Breakthrough Therapy Designation (BTD) process by China's Center for Drug Evaluation (CDE). This designation targets the treatment of adult plexiform neurofibromas associated with symptomatic and inoperable neurofibromatosis type I (NF1). The inclusion in the BTD process is expected to accelerate the drug launch, potentially benefiting patients sooner.

Previously, Phase I clinical trials for TQ-B3234 demonstrated significant efficacy. Results presented at the 2025 ASCO Annual Meeting indicated that 96.7% of NF1-PN subjects experienced tumor shrinkage, with 36.7% achieving partial response. An exploratory analysis in cNF patients showed 100% tumor shrinkage. TQ-B3234 also exhibited good tolerability in adult NF1 patients.

NF1, an autosomal dominant disorder affecting an estimated 5 per million Chinese newborns, often leads to plexiform neurofibromas in 30-60% of patients. While surgery is currently the main treatment, MEK inhibitors are preferred for inoperable cases. With the phase III clinical study for adult PN treatment already approved by the CDE, TQ-B3234 aims to address the unmet medical need for effective adult PN therapies in China.