FDA Designates ERAS-801, a CNS-Penetrant EGFR Inhibitor, as Orphan Drug for Malignant Glioma Treatment

Friday, June 23, 2023

ERASCA, a clinical-stage precision oncology company focused on developing therapies for RAS/MAPK pathway-driven cancers, has announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ERAS-801 for the treatment of malignant glioma, including glioblastoma (GBM). ERAS-801 is an orally bioavailable EGFR inhibitor with significant central nervous system (CNS) penetration, as demonstrated in preclinical animal studies.

Orphan Drug Designation is granted by the FDA to investigational therapies targeting rare medical diseases or conditions affecting fewer than 200,000 people in the United States. This designation provides several benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees, and the potential for seven years of post-approval marketing exclusivity.

Jonathan E. Lim, M.D., Chairman, CEO, and Co-founder of ERASCA, highlighted the challenges associated with current EGFR inhibitors for treating malignant glioma, emphasizing their limited CNS penetration and minimal activity against GBM-specific EGFR alterations. He expressed optimism about ERAS-801's potential to overcome these challenges, citing its broad activity against oncogenic and wildtype EGFR, high CNS penetration, and demonstrated efficacy in diverse EGFR-driven glioma models.

The ODD for ERAS-801 underscores the significance of innovation for GBM patients and the therapeutic potential of ERAS-801 as a targeted treatment option. It follows the earlier Fast Track Designation granted by the FDA, emphasizing the urgency of finding new treatments for this patient population. ERASCA anticipates reporting initial monotherapy data from the Phase 1 THUNDERBOLT-1 trial for recurrent GBM patients in the second half of 2023.

ERAS-801 was developed by a team of renowned cancer researchers, including Dr. Michael Jung, Dr. Timothy Cloughesy, and Dr. David Nathanson.

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