OncLive’s OncFive presents five top oncology stories of the week.
The FDA approved narsoplimab‑wuug (Yartemlea) for adults and pediatric patients aged 2 years and older with hematopoietic stem cell transplant–associated thrombotic microangiopathy (TA‑TMA). Approval was based on single‑arm study and expanded access data showing a 61% complete response rate among evaluable patients, with consistent responses across adult and pediatric cohorts. Treatment was associated with improved platelet counts, lower lactate dehydrogenase, better organ function, increased transfusion independence and an approximately 73% 100‑day survival from TMA diagnosis. This is the first FDA‑approved therapy for TA‑TMA.
A new drug application has been submitted to the FDA for bezuclastinib (CGT0486) for nonadvanced systemic mastocytosis after positive results from the phase 2 SUMMIT trial. Bezuclastinib produced significant improvements in total symptom score at week 24 versus placebo and higher rates of clinically meaningful symptom reduction, and demonstrated strong biologic activity with more than 95% of evaluable patients achieving at least a 50% reduction in serum tryptase levels.
China’s National Medical Products Administration approved ipilimumab N01 (IBI310; Tabosun) in combination with sintilimab (Tyvyt) as neoadjuvant therapy for patients with stage IIB–III resectable microsatellite instability–high/mismatch repair–deficient colon cancer. The approval was supported by phase 3 NeoShot data showing an 82% pathological complete response rate with the combination versus surgery alone without increased surgical risk; earlier phase 1b data also showed higher pCR rates with the combination versus sintilimab monotherapy. This marks the first approval of a CTLA‑4 antibody in the neoadjuvant colon cancer setting.
Japan’s Ministry of Health, Labour and Welfare approved tafasitamab‑cxix (Minjuvi) plus rituximab and lenalidomide for adults with relapsed or refractory follicular lymphoma. The decision was based on the phase 3 inMIND trial, which reported a median progression‑free survival of 22.4 months with the tafasitamab combination versus 13.9 months with placebo plus rituximab and lenalidomide (hazard ratio 0.43); independent review found durable PFS benefit and median PFS was not reached in the tafasitamab arm. The approval provides a chemotherapy‑free, dual CD19/CD20‑targeted option for relapsed/refractory disease in Japan.
December 2025 saw multiple FDA oncology decisions, including full approval of pirtobrutinib (Jaypirca) for relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma based on BRUIN CLL‑321, and clearance of lisocabtagene maraleucel (Breyanzi) for relapsed/refractory marginal zone lymphoma based on TRANSCEND‑FL. Other approvals included niraparib plus abiraterone (Akeega) for BRCA2‑mutated metastatic castration‑sensitive prostate cancer (AMPLITUDE) and fam‑trastuzumab deruxtecan‑nxki (Enhertu) plus pertuzumab (Perjeta) for frontline HER2‑positive metastatic breast cancer (DESTINY‑Breast09). The agency also expanded access to subcutaneous formulations of amivantamab and hyaluronidase‑lpuj (Rybrevant Faspro) and to mosunetuzumab, improving treatment convenience without compromising efficacy. Collectively, these decisions underscore continued momentum toward precision oncology, novel delivery platforms and earlier use of targeted and cellular therapies.
Leave a Reply