Key Takeaways:
- FDA approved AbbVie's Decnupaz for ultra-rare blood cancer BPDCN
- Frontline patients achieved 69% composite complete response rate
- First ADC for BPDCN that can be initiated in an outpatient setting
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AbbVie's Decnupaz wins FDA approval with 69% response rate in rare cancer
The FDA on Wednesday approved AbbVie's Decnupaz for an ultra-rare blood cancer, with 69% of previously untreated patients achieving a composite complete response.
"These strong, durable response results offer hope to BPDCN patients with limited treatment options," Naveen Pemmaraju, MD, principal investigator and professor at the University of Texas MD Anderson Cancer Center, said in a statement.
The approval is based on the CADENZA trial, a single-arm open-label study of 84 adults with blastic plasmacytoid dendritic cell neoplasm. The study population included 33 patients with previously untreated disease — 22 with de novo BPDCN and 11 with a co-existing hematologic malignancy — and 51 with relapsed or refractory disease. Among the frontline group, 69% met the primary endpoint of composite complete response, defined as complete remission plus clinical complete remission, with a median duration of 9.7 months after a median follow-up of 21.5 months. In the relapsed or refractory group, 15.7% achieved response with a median duration of 9.2 months after a median follow-up of 24.1 months.
BPDCN affects an estimated 500 to 1,000 patients annually in the US and is highly aggressive, involving skin, bone marrow and lymph nodes. The CD123 antigen is overexpressed in BPDCN cells, providing the rationale for Decnupaz, an anti-CD123 antibody-drug conjugate. It is the first ADC for BPDCN that can be initiated in an outpatient setting, a potential advantage over tagraxofusp (Elzonris), the only other FDA-approved therapy for the disease.
The prescribing information includes a boxed warning for hepatotoxicity, including hepatic veno-occlusive disease, as well as warnings for infusion-related reactions, edema, sulfite allergic reactions and embryo-fetal toxicity. Decnupaz is administered intravenously once every three weeks.
AbbVie shares rose more than 1% in afternoon trading following the announcement, according to Reuters. The stock has gained roughly 12% year to date, outperforming the S&P 500's health care sector.
The approval expands AbbVie's oncology portfolio with its first antibody-drug conjugate for a blood cancer. Investors will watch for initial sales data and potential label expansion into other CD123-positive malignancies.
This article is for informational purposes only and does not constitute investment advice.
[1] U.S. FDA Approves DECNUPAZTM (pivekimab sunirine-pvzy) for Treatment of Adult Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm, an Ultra-Rare and Aggressive Blood Cancer With Limited Treatment Options[2] Decnupaz Approved for Rare Blood Cancer[3] US FDA approves AbbVie's rare blood cancer drug[4] ABBV stock gains on winning FDA approval for rare blood cancer drug
