The drug was previously granted the same designation in March 2016 for the treatment of autosomal recessive polycystic kidney disease (ARPKD).
EGFR, a cell surface receptor, has a gene that is amplified in more than half of all gliomas. Studies have shown other EGFR inhibitors to have poor brain penetration, which limits their abilities to reach and successfully treat brain tumors. Different from other EGFR inhibitors, tesevatinib has been observed to be highly blood-brain barrier penetrant, reaching equivalent concentrations in the brain and the blood.
The company is conducting an ongoing Phase 2 clinical trial of tesevatinib in NSCLC with EGFR-activating mutations that has metastasized to the brain or meninges surrounding the central nervous system. The drug is also being investigated in a separate study assessing it in patients with recurrent glioblastoma. The Company is also developing tesevatinib for the treatment of autosomal dominant polycystic kidney disease (ADPKD) and ARPKD and expects to initiate clinical trials in these indications in Q3 2017.
“We are encouraged by tesevatinib’s potential ability to cross the blood-brain barrier in humans, which may lead to meaningful clinical activity against brain tumors,” said Harlan W. Waksal, MD, President and Chief Executive Officer at Kadmon Holdings in an earlier press release.