Orphan Status for BRAF Inhibitor

FDA has granted ABM Therapeutics an orphan drug designation for ABM-1310, a novel small molecule BRAF inhibitor for treating patients with glioblastoma (GBM) who have the BRAF V600 mutation. The company says the drug has BRAF-mutation selectivity, high water solubility, and high blood-brain barrier permeability.

ABM-1310 is in Phase I studies at multiple clinical sites in the U.S. and China for BRAF V600-mutant advanced solid tumors, according to ABM Therapeutics. Interim results from its U.S. Phase 1 study were presented in June at the American Society of Clinical Oncology annual meeting, it says, adding that data demonstrated “promising anticancer activity and good safety profile in patients with advanced BRAF V600 mutant solid tumors including primary brain tumors such as GBM and other gliomas.” A new Phase 1 clinical trial specifically targeting GBM has recently begun in China.