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.

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.