FDA approves first cancer drug based on ӳý science
Collaboration between ӳý and Bayer leads to new treatment for a hard-to-treat type of lung cancer.
Highlights
- The new medicine, sevabertinib, is a pill approved for a type of lung cancer that previously had few treatment options.
- Genetic discoveries from ӳý scientists laid the foundation for the development of the drug.
- This FDA approval is an important milestone for the ӳý, demonstrating that research by academic scientists and close collaborations with industry can lead to benefits for patients.
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The US Food and Drug Administration has approved the first cancer drug based on discoveries from ӳý scientists. The drug, sevabertinib, was developed by Bayer Healthcare Pharmaceuticals in close collaboration with the ӳý, and is the first FDA-approved medicine from the long-standing ӳý-Bayer oncology research alliance.
Sevabertinib is a new oral treatment option for a type of non-small-cell lung cancer, the most common type of lung cancer. The medicine is a pill that patients can take at home and is approved for adult patients with certain HER2 mutations in their tumors and who have already received chemotherapy or immunotherapy. This translates to roughly 4,000 to 8,000 lung cancer patients in the US every year who could potentially benefit from this new drug. Many of these patients are women, including younger women who have never smoked.
The drug's approval is based on data from Bayer’s Phase I/II clinical trial, which found that over 70 percent of the patients studied in one cohort saw their tumors shrink or disappear. Many patients experienced profound and durable responses.
This achievement is the result of discoveries made by ӳý scientists two decades ago and of the collaboration between ӳý and Bayer Pharmaceuticals, which began in 2013. The team's clinical success demonstrates that long-term academic-industry partnerships can close the gap between biological discoveries and new medicines that improve health.
"The whole idea of the ӳý 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 Matthew Meyerson, a lead cancer biologist on the project, institute member at the ӳý, the Charles A. Dana Chair in Human Cancer Genetics at Dana-Farber Cancer Institute, and professor of genetics and medicine at Dana-Farber and Harvard Medical School. "Today, with our industry partners at Bayer, we're now able to see the fruits of these efforts in a new drug that we hope can help many patients by extending their lifespan and improving their quality of life."
New option for lung cancer patients
Lung cancer is the leading cause of cancer-related deaths in the US, and non-small-cell lung cancer (NSCLC) accounts for more than 85 percent of lung cancer cases. Up to 4 percent of NSCLC cases carry a HER2 gene mutation, which causes the HER2 protein to become overactive and spur abnormal cell growth.
Sevabertinib halts the activity of HER2 proteins with certain mutations, inhibiting the growth of tumors. Approved as a second-line treatment, it may serve a patient population that until recently had no remaining options after chemotherapy or immunotherapy for treating their cancer.
The FDA granted sevabertinib a in 2024, signifying that the compound may provide substantial improvement over existing therapies in an area of high unmet medical need. In 2025, sevabertinib was additionally , meaning the FDA would aim to take action on an application within six months (compared to ten months under standard review).
A research success story
This milestone began with work led by Meyerson and Heidi Greulich, an institute scientist and senior group leader in the Cancer Program at the ӳý.
In 2005, the ӳý team reported that some lung cancer patients who didn't respond to existing drugs had a mutation in their tumors known as an “exon 20 insertion” — an extra bit of DNA tucked in at a specific location in the gene EGFR. Developing a drug to target this mutation was one of the first projects for the ӳý-Bayer alliance when it launched in 2013, and the work soon evolved into creating a drug to target a similar mutation in HER2, a gene closely related to EGFR that’s involved in lung and other cancers.
"We've been collaborating closely with the Bayer team on sevabertinib and its precursor compounds for 12 years now,” said Greulich. “It really shows how the power of persistence can be brought to bear on difficult problems to help patients."
A Phase III clinical trial is continuing to evaluate sevabertinib as a first-line treatment option for patients with NSCLC carrying HER2 mutations. The drug is also being studied in patients with other metastatic or unresectable solid tumors, beyond lung cancer, that have HER2 mutations.
"We're in an extraordinary time for the development of new medicines to treat cancer patients," said Meyerson. "And I think that, with our industry partners, we have a huge opportunity to repeat this success not once, not twice, but many, many times over."
