FDA approves new oral treatment for non-small-cell lung cancer

Over twenty years ago, scientists at the Broad Institute, including Matthew Meyerson, MD, PhD (Damon Runyon Fellow ’95-’98), William R. Sellers, MD (Damon Runyon Clinical Investigator '01-'05, Board Member), and Todd R. Golub, MD (Innovation Award Committee Member, Board Member), set out to understand why some patients with lung cancer did not respond to existing treatments. Now, these decades of work have paid off: the FDA has approved a new oral treatment for non-small-cell lung cancer, the most common form of the disease.

Approved for adults whose tumors carry an HER2 mutation and who have already undergone a first-line treatment, the pill known as sevabertinib offers a new option for a patient population, including younger women who have never smoked, for whom effective treatments have long been limited.

The road to this milestone began with a paper published in 2005, in which Broad researchers identified a mutation in the gene EGFR that confers drug resistance to lung cancer cells. Based on this work, researchers developed a drug to target a similar mutation in HER2, a gene closely related to EGFR that plays a role in lung and other cancers.

“The whole idea of the Broad Institute when it was founded more than 20 years ago was to bring the power of human genome science and high-throughput research to solving human disease,” said Dr. Meyerson. “We’re now able to see the fruits of those efforts.”

The FDA recognized the promise of sevabertinib by granting it Breakthrough Therapy designation and Priority Review, underscoring its potential to address a critical unmet need. Ongoing Phase III trials are now testing the drug as a first-line treatment, and researchers are exploring its use in other HER2-mutant cancers beyond lung cancer.