Decnupaz is the first antibody-drug conjugate for blastic plasmacytoid dendritic cell neoplasm, an ultra-rare and aggressive blood cancer.
The FDA has cleared the use of AbbVie’s antibody-drug conjugate for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm. The drug will carry the brand name Decnupaz.
Decnupaz is the first and so far only antibody-drug conjugate (ADC) approved for this indication, according to the company’s Wednesday news release. The approval also marks AbbVie’s first ADC entry in the blood cancer space.
The FDA’s decision was backed by data from the Phase 1/2 CADENZA study, which enrolled 33 treatment-naive patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) and 51 patients with relapsed or refractory BPDCN. None of the patients had signs of central nervous system disease.
In treatment-naive patients, Decnupaz elicited a 69.7% complete remission or clinical complete remission (CR/CRc) rate at a median follow-up of 21.5 months, according to the FDA’s announcement of the drug’s approval. Meanwhile, in those who had prior histories of treatment, the CR/CRc rate was 15.7% at a median of 24.1 months follow-up.
As for safety, AbbVie on Wednesday flagged side effects such as edema, fatigue, musculoskeletal pain, hemorrhage, nausea and diarrhea, all of which occurred in at least 20% of treated patients.
The FDA also slapped Decnupaz with a boxed warning for hepatotoxicity, flagging patients and prescribers to the risk of blockages in the small veins in the liver, which could become life-threatening and lead to death.
Affecting 500 to 1,000 patients in the U.S. per year, BPDCN is a rare form of leukemia that afflicts the bone, skin and lymph nodes. The disease manifests primarily as skin lesions, which typically vary in shape, size and color. While the initial presentations seem harmless, BPDCN is aggressive and can quickly spread throughout the body, often causing blood-related complications such as anemia and thrombocytopenia.
Decnupaz works by binding to CD123, a key biomarker found on the surface of BPDCN and other blood cancer cells. The therapy carries an indolinobenzodiazepine payload, which target and damage DNA when released inside a malignant cell, in turn destroying the cancer.
Aside from BPDCN, AbbVie is also studying Decnupaz for acute myeloid leukemia, for which the asset is in mid-stage development.
