FDA Orphan Drug Designation for ERAS-801 for the Treatment of Malignant Glioma

“GBM is an aggressive malignancy afflicting approximately 37,000 patients annually in the United States and Europe. Currently approved EGFR inhibitors are limited by insufficient CNS penetration to treat GBM and minimal activity against GBM-specific EGFR amplifications, mutations, and other molecular alterations, which contribute to high rates of relapse and a five-year survival rate below 10%,” said Jonathan E. Lim, M.D., Erasca’s chairman, CEO, and co-founder. “Receiving ODD recognizes both the importance of innovation for patients with GBM and the therapeutic potential of ERAS-801 to provide a targeted treatment option for these patients, who have a poor prognosis. This ODD follows the earlier Fast Track Designation granted to ERAS-801 by the FDA and underscores the urgency of finding new treatments for this patient population. The broad activity against both oncogenic and wildtype EGFR, high CNS penetration, and demonstrated ability to improve outcomes in over 90% of diverse EGFR-driven patient-derived glioma models support the potential for ERAS-801 to overcome current challenges with existing therapies. We anticipate reporting initial monotherapy data for ERAS-801 from the Phase 1 THUNDERBBOLT-1 trial in patients with recurrent GBM in the second half of 2023.”