Pevonedistat hydrochloride is primarily used in the field of oncology as an investigational drug targeting cancer treatment. It functions as a NEDD8-activating enzyme (NAE) inhibitor, which disrupts the NEDD8 pathway essential for the degradation of certain proteins that regulate cell cycle and survival. By inhibiting this pathway, it promotes the accumulation of these proteins, leading to cancer cell death. This mechanism is particularly explored in the treatment of hematologic malignancies such as acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). Additionally, it is being studied in combination with other chemotherapeutic agents to enhance efficacy and overcome drug resistance in various solid tumors. Its application is still under clinical trials to determine safety, dosage, and long-term outcomes.