FDA OKs first targeted therapy for lung cancer with certain type of EGFR mutations

About 2% to 3% of patients with nonsmall cell lung cancer have EGFR exon 20 insertion mutations targeted by newly approved Rybrevant.

FDA cleared amivantamab-vmjw (Rybrevant, Janssen) as the first treatment for adult patients with non-small cell lung cancer (NSCLC) whose tumors have specific types of genetic mutations.

The genetic mutations, called epidermal growth factor receptor (EGFR) exon 20 insertion mutations, are detected by an FDA-approved companion test, Guardant360 CDx (Guardant Health).

Approximately 2% to 3% of patients with non-small cell lung cancer have EGFR exon 20 insertion mutations, which are a group of mutations on a protein that causes rapid cell growth, according the FDA's press release about the approval. EGFR exon 20 insertion mutations are the third most common type of EGFR mutation.

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“Today’s FDA approval is an important development for people living with non-small cell lung cancer with exon 20 insertion mutations who, until now, have had no approved treatment options to target their disease,” said Jill Feldman, co-founder of the EGFR Resisters, an advocacy patient group, in a Janssen press release. “We are excited by the promise this new treatment option brings to people with this particular type of lung cancer and their families.”

Advances in precision oncology continue to facilitate drug development, allowing diseases like lung cancer to be subset into biomarker-defined populations appropriate for targeted therapies, said Julia Beaver, MD, chief of medical oncology in the FDA’s Oncology Center of Excellence and acting deputy director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, in a press release.

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FDA’s accelerated approval was based on a study of the medication’s efficacy in 81 patients with non-small cell lung cancer and EGFR exon 20 insertion mutations whose disease had progressed on or after platinum-based chemotherapy.

The overall response rate of patients receiving Rybrevant was 40%. The median duration of response was 11.1 months, with 63% of patients having a duration of response of 6 months or more.

FDA previously granted Breakthrough Therapy Designation (BTD) to Rybrevant in March 2020 and initiated a Priority Review of the Biologics License Application (BLA) in December 2020.

Read more: Lung Cancer Drug Snags Priority Review