FDA Approves New Drug for Treating Most Common Type of Lung Cancer, Based on Broad Institute Research

The Food and Drug Administration granted accelerated approval on Wednesday for a new drug developed by researchers at the Broad Institute that treats non-small cell lung cancer — the most common type of lung cancer, which had until recently few treatments.

The medicine, called sevabertinib, comes in the form of a pill and helps treat adults with certain HER2 gene mutations, a group that represents between two to four percent of NSCLC patients in the United States, or roughly 4,000 to 8,000 people each year.

The discovery was the culmination of a 12-year-long partnership between the Broad Institute, a biomedical research organization affiliated with Harvard and MIT, and Bayer Healthcare Pharmaceuticals, a global healthcare company.

“Up until a couple of years ago, there was nothing available in terms of targeted therapy for these patients,” said Heidi E. Greulich, a senior group leader at the Broad Institute who undertook a postdoctoral fellowship at Harvard.

But now there is. The researchers conducted clinical trials and found that when treated with sevabertinib, more than 70 percent of patients had their tumors shrink or even disappear. Clinical trial data were presented in October at a Berlin conference and in the pages of the New England Journal of Medicine.

Based on the strong findings in the first two rounds of clinical trials, the FDA approved the drug for distribution — even before the researchers had completed the third phase of clinical trials.

The development of sevabertinib started more than two decades ago.

In 2003, Greulich and Harvard Medical School professor Matthew L. Meyerson ’85, who is an institute member at the Broad, began work on developing treatments that could address a specific mutation in the EFGR gene, known as an “exon-20 insertion.”

The researchers quickly realized that they could apply their research to the HER2 gene, which shared a similar mutation to the EFGR gene and which is targeted by sevabertinib.

The process of developing what would become sevabertinib was not without challenges. Bayer was close to shutting down the project when some initial models of the molecule that comprises the drug were unstable, Greulich said.

But the company stuck by the researchers, contributing millions of compounds and providing the majority of the funding for the project. The Exon 20 Group, a nonprofit dedicated to treating lung cancer patients with exon-20 insertions, also contributed some funds to the effort.

Sevabertinib is not the only recent drug for treating non-small cell lung cancer. In 2022, the FDA approved the drug trastuzumab for treating patients with NSCLC. But the drug had a drawback: it required patients to visit the hospital for an infusion.

Three years later, in 2025, the FDA also approved zongertinib, another drug for NSCLC patients. Greulich said that though her team has been “in a little bit of a race” with the researchers developing zongertinib, both drugs add value by providing more options to patients with lung cancer.

“The mechanisms of resistance to these two drugs are anticipated to be different, so there should still be space for both drugs. And I think that the beauty of it is it gives the patients and their prescribing physicians more options,” Greulich added.

Sevabertinib is another landmark in decades of rapid advances in cancer treatments, according to Meyerson, and could indicate that new treatment innovations are soon to come.

“I feel like this advance that we have with this particular drug is one in many steps in that journey to improved care for cancer patients,” Meyerson said.