DAY101 Wins Orphan Drug Designation By European Commission

Release Date: 30-Sep-2021

European Commission has granted orphan drug designation for DAY101, for the treatment of glioma. The most common brain tumor diagnosed in children is Pediatric low-grade glioma (pLGG), accounting for 30% andndash; 50% of all central nervous system tumors. It affects the health of a child in many different ways depending on tumor size and location, including vision loss and motor dysfunction.


It is given to investigational therapies that have the power to treat rare and life-threatening diseases that affect less than five in 10,000 patients in the European Union. It provides some benefits to companies, like clinical protocol advice, fee reductions, and ten years of protection from competition following regulatory approval.


DAY101 is an oral, brain-penetrant, highly-selective type II pan-RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway. Studies have shown DAY101 has high brain distribution and exposure in comparison to other MAPK pathway inhibitors, thus potentially benefiting patients with primary brain tumors or brain metastases of solid tumors. DAY101 is a type II RAF inhibitor found to selectively inhibit both monomeric and dimeric RAF kinase, which may broaden its potential clinical application to treat an array of RAF-altered tumors. Also, drug DAY101 has received Orphan Drug designation from the FDA for the treatment of malignant glioma and orphan designation from the European Commission for the treatment of glioma.

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