Erasca granted FDA Orphan Drug Designation for malignant glioma treatment

Erasca, a clinical-stage precision oncology company singularly focused on discovering, developing, and commercializing therapies for patients with RAS/MAPK pathway-driven cancers, has been granted Orphan Drug Designation (ODD) by the United States Food and Drug Administration (FDA) for ERAS-801 for the treatment of malignant glioma, which includes glioblastoma (GBM).

ERAS-801 is an orally bioavailable, small molecule EGFR inhibitor that exhibited substantial central nervous system (CNS) penetration in preclinical animal studies.

Per FDA regulations, ODD is granted by the FDA to investigational therapies addressing rare medical diseases or conditions affecting less than 200,000 people in the United States. Orphan drug status provides 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.

“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.”

ERAS-801 was designed and developed by a renowned team of cancer researchers—Michael Jung, Ph.D., Timothy Cloughesy, M.D., and David Nathanson, Ph.D.

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