The U.S. Food and Drug Administration (FDA) has made two important decisions in cancer treatment this week. First, they approved a new drug called Decnupaz (pivekimab sunirine) for people with a very rare and aggressive blood cancer called blastic plasmacytoid dendritic cell neoplasm (BPDCN). This is the first targeted therapy approved specifically for BPDCN, which is difficult to treat and often comes back even after standard chemotherapy and stem cell transplants. In clinical trials, Decnupaz helped 70% of newly diagnosed patients and 16% of patients whose cancer had returned or did not respond to other treatments achieve a strong response. However, it does have a warning label because it can cause liver problems. Decnupaz will compete with another drug, Elzonris (tagraxofusp), which was approved in 2018 as the first targeted treatment for BPDCN. One advantage of Decnupaz is that it can be given to patients on an outpatient basis, meaning they don’t need to stay in the hospital for treatment.
Meanwhile, the FDA has delayed its decision on AstraZeneca’s new breast cancer drug, camizestrant. This drug is designed to treat advanced breast cancer in postmenopausal women whose cancer is hormone receptor-positive (HR+) and HER2-negative, and has a specific genetic mutation called ESR1. The delay comes after an advisory committee reviewed the drug and voted six to three against recommending it, because they felt it did not show enough benefit for patients. The FDA can still approve the drug even if the committee does not recommend it. Camizestrant is a next-generation oral therapy that blocks estrogen receptors completely, which could help overcome resistance to current hormone therapies like aromatase inhibitors. It is being tested alongside a cyclin-dependent kinase (CDK)4/6 inhibitor, which is a standard treatment for this type of breast cancer.
Additionally, AstraZeneca recently received FDA approval for another drug, Datroway (datopotamab deruxtecan), for the first-line treatment of triple-negative breast cancer (TNBC). This is the first drug in its class to be approved for TNBC, giving patients a new option before they try other treatments. AstraZeneca was able to bring this drug to market ahead of Gilead, which is still waiting for a decision on its similar drug, Trodelvy (sacituzumab govitecan), for the same type of breast cancer.