Benzinga | Jun 21, 2021 07:04AM EDT

Day One Receives Orphan Designation from the European Commission for DAY101 for the Treatment of Glioma

Day One Biopharmaceuticals, a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for patients of all ages with genetically defined cancers, today announced that the European Commission has granted orphan designation for the Company's lead product candidate, DAY101, for the treatment of glioma. The designation was based on a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products.

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. Day One is conducting a pivotal Phase 2 clinical trial (FIREFLY-1) of DAY101 in pediatric, adolescent and young adult patients with recurrent or progressive low-grade glioma (pLGG) harboring a known BRAF alteration.

"Orphan designation for DAY101 from the European Commission exemplifies the urgent medical need for additional treatment options for glioma and builds upon the previous Breakthrough Therapy and Orphan Drug designations from the U.S. Food and Drug Administration for DAY101," said Davy Chiodin, PharmD, chief development officer of Day One. "We look forward to continue enrolling patients in the pivotal Phase 2 FIREFLY-1 study which has the potential to make DAY101 the first approved therapy for patients with pLGG."