FDA Grants FDA Fast Track Designation for Erasca’s ERAS-801 in Patients with Glioblastoma

Rare Daily Staff

The U. S. Food and Drug Administration granted Fast Track designation to Erasca’s ERAS-801 for the treatment of adult patients with glioblastoma with epidermal growth factor receptor (EGFR) gene alterations.

Glioblastoma (GBM) is an aggressive malignancy with high rates of relapse and a five-year survival rate below 10 percent. While over half of GBM cases are driven by EGFR alterations and/or amplifications, there are no approved EGFR inhibitors for the treatment of GBM due to the lack of sufficient brain penetration to treat primary brain tumors as well as lack of activity against EGFR alterations observed in GBM.

ERAS-801, an orally bioavailable, small molecule EGFR inhibitor, was specifically designed to have high CNS penetration and broad activity against both oncogenic and wildtype EGFR.

“Receiving Fast Track designation from the FDA underscores the serious unmet medical need in patients with GBM and reinforces the promise that ERAS-801 may offer as a differentiated treatment option,” said Jonathan Lim, chairman, CEO, and co-founder of Erasca. “We look forward to working closely with the FDA to expedite clinical development of ERAS-801 for these patients and anticipate reporting initial monotherapy data from the phase 1 THUNDERBBOLT-1 trial in recurrent GBM (rGBM) in the second half of 2023.”

Fast Track designation is designed to help drugs reach patients faster by facilitating the development and expediting the review of drugs with the potential to fill an unmet medical need by treating a serious or life-threatening condition. Programs that receive FTD benefit from early and frequent interactions with the FDA during the clinical development process and, if relevant criteria are met, the FDA may consider reviewing portions of a marketing application before the sponsor submits the complete application.

In animal models, ERAS-801 had a brain-to-plasma that was up to four times higher than approved EGFR inhibitors, suggesting that approximately 100 percent of the free drug in plasma is able to cross the blood-brain barrier.

At clinically relevant exposures across 30 patient-derived GBM models that are intended to represent the heterogeneity of GBM, ERAS-801 demonstrated a survival benefit in 13 out of 14 EGFR mutant and/or amplified models and had statistically significantly higher brain penetrance and prolonged survival compared to approved EGFR tyrosine kinase inhibitors. ERAS-801 is currently being evaluated as a monotherapy in THUNDERBBOLT-1, an ongoing phase 1 trial in patients with rGBM.

Photo: Jonathan Lim, chairman, CEO, and co-founder of Erasca