In 2024, the U.S. Food and Drug Administration (FDA) approved 11 drugs based on clinical trials in which MSK played a pivotal role.
These approvals expand treatment options for a range of cancers, including subtypes of brain, lung, breast, thyroid, and colorectal cancer, and span new uses for existing drugs, more effective drug combinations, engineered cell therapies, and novel classes of targeted therapies.
Together, they exemplify the kind of bench-to-bedside advances that are possible at MSK, where basic and clinical research are united.
FDA approvals in 2024, in chronological order:
- For metastatic non-small cell lung cancer caused by a genetic error called METex14, the FDA granted approval to the targeted therapy tepotinib (Tepmetko®) on February 15, 2024. Tepotinib was approved based on the findings from a multicenter international phase 2 clinical trial known as VISION. MSK thoracic oncologist Paul Paik, MD, Clinical Director of MSK’s Thoracic Oncology Service, was lead author of the study.
- For a rare blood cancer called mantle cell lymphoma, the FDA approved the CAR T treatment lisocabtagene maraleucel (Breyanzi®). The decision on May 30, 2024, was based on results from a clinical trial led by MSK hematologist-oncologist Lia Palomba, MD.
- For treating adult and pediatric patients with advanced or metastatic thyroid cancers with RET mutations, the FDA granted full approval to the targeted therapy selpercatinib (Retevmo®) on June 12, 2024. MSK head and neck medical oncologist Eric Sherman, MD, was one of the leaders of the clinical trial studying selpercatinib for thyroid cancer.
- For treating cancers anywhere in the body caused by gene changes called NTRK fusions, a targeted drug called repotrectinib (AugtyroTM) received accelerated approval from the FDA on June 13, 2024. The FDA action was based on a global clinical trial led by early drug development specialist Alexander Drilon, MD, Chief of MSK’s Early Drug Development Service.
- For people with advanced colorectal cancer caused by a gene mutation called KRAS-G12C, the FDA granted accelerated approval for the targeted therapy adagrasib (Krazati®) in combination with cetuximab (Erbitux®) on June 21, 2024. The trial that resulted in the approval was led by MSK gastrointestinal medical oncologist and early drug development specialist Rona Yaeger, MD.
- For the treatment of adults with a rare soft tissue cancer called synovial sarcoma, the FDA granted accelerated approval for the immunotherapy afamitresgene autoleucel (Tecelra®) on August 2, 2024. The clinical trial that resulted in the drug’s approval was led by MSK sarcoma specialist and immunotherapy expert Sandra D’Angelo, MD.
- For brain tumors called low-grade diffuse gliomas that carry an IDH gene mutation, the FDA approved the drug vorasidenib (Voranigo®) on August 6, 2024. MSK’s Ingo Mellinghoff, MD, FACP, Chair of the Department of Neurology, led the clinical trial demonstrating the drug’s potential.
- For certain breast cancers, the FDA approved a new three-drug combination. The treatment combines a new targeted drug, inavolisib (Itovebi®), with two older drugs, fulvestrant (Faslodex®) and palbociclib (Ibrance®). The approval on October 10, 2024, was based on a trial co-led by MSK medical oncologist and breast cancer specialist Komal Jhaveri, MD.
- For children and adults with leukemia that has come back after treatment and carries a molecular change called a KMT2A translocation, the FDA approved the drug revumenib (Revuforj®). The decision on November 15, 2024, was based on results of a clinical trial led by Eytan Stein, MD, Chief of the Leukemia Service at MSK.
- For treating advanced and metastatic bile duct cancer (cholangiocarcinoma) and other biliary tract cancers with high levels of HER2, the FDA granted accelerated approval to zanidatamab (Ziihera®). The approval on November 20, 2024, was based on a trial co-led by MSK gastrointestinal medical oncologist and early drug development specialist James J. Harding, MD.
- For treating patients with advanced pancreatic cancer or non-small cell lung cancer that has an alteration called an NRG1 gene fusion, the FDA granted accelerated approval to zenocutuzumab (Bizengri®). The December 4, 2024, decision was based on a phase 2 clinical trial led by MSK early drug development specialist and gynecologic medical oncologist Alison Schram, MD.
Note: The FDA uses the term “accelerated approval” when granting approval for drugs that treat diseases lacking effective treatments. These criteria are slightly different than for a standard approval. Also, the term “full approval” applies to drugs that had previously been granted “conditional approval.”