Erasca’s ERAS-801 will be used to treat malignant glioma, including glioblastoma. Credit: Nemes Laszlo / Shutterstock.com.

Erasca has obtained orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for ERAS-801 to treat malignant glioma, including glioblastoma (GBM).

ERAS-801 is an orally bioavailable, small-molecule epidermal growth factor receptor (EGFR) inhibitor that demonstrated penetration of the substantial central nervous system (CNS) in preclinical animal studies.

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The therapy is being assessed as a monotherapy in an ongoing Phase I THUNDERBBOLT-1 trial in recurrent GBM (rGBM) patients.

The trial has been designed to assess ERAS-801’s preliminary efficacy, safety and tolerability as a monotherapy in rGBM patients.

The dose escalation portion of the trial will determine the recommended dose to be used, and will further assess safety and efficacy.

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Erasca chairman, CEO and co-founder Jonathan Lim stated: “GBM is an aggressive malignancy afflicting approximately 37,000 patients annually in the United States and Europe.

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“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%.

“Receiving ODD recognises 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.”

The company expects to report initial monotherapy data from the THUNDERBBOLT-1 trial in the second half of 2023.

In May 2023, the regulator granted fast track designation (FTD) to ERAS-801 to treat GBM in adult patients with EGFR gene alterations.