🔬🩸 Hematology Updates: Myeloma Advances, Menin Inhibitor Momentum, and Orphan Drug Wins
From powerful long-term data in multiple myeloma and priority reviews for targeted AML therapies to orphan drug milestones in sickle cell disease—this week’s hematology briefing delivers cutting-edge clinical results and regulatory accelerations driving progress in blood cancer and rare hematologic conditions.
💡 Key Highlights This Week:
✅ DARZALEX FASPRO achieves greater than 95 percent 4-year PFS in newly diagnosed multiple myeloma, with sustained MRD negativity over 24 months
🧪 IGI’s trispecific ISB 2001 reports 79% ORR and 30% CR/sCR in heavily pretreated relapsed/refractory multiple myeloma
🚀 Menin inhibitor ziftomenib receives FDA Priority Review for NPM1-mutant AML, with a PDUFA date set for November 30, 2025
🧬 Selinexor shows 52% deep molecular responses in pediatric R/R AML targeting high-risk fusions and rearrangements
🇪🇺 Calquence plus venetoclax earns EU approval as first all-oral, fixed-duration frontline CLL regimen
🧪 Halia completes Phase 2a enrollment in low-risk MDS trial targeting NEK7-NLRP3 inflammasome; topline data expected late 2025
🔔 Cellectar gains FDA Breakthrough Therapy status for iopofosine I-131 in Waldenström’s macroglobulinemia, showing 84% ORR
🏥 Estrella expands U.S. trial sites for CD19 T-cell therapy EB103 using ARTEMIS platform in R/R B-cell NHL
📊 ADCETRIS BrECADD approved in EU for frontline advanced Hodgkin lymphoma, showing superior safety over eBEACOPP
🧬 Beam’s base-edited BEAM-101 receives FDA Orphan Drug Designation for sickle cell disease with robust HbF induction
💊 Sanofi’s rilzabrutinib wins its fourth orphan tag, now for sickle cell disease, based on anti-inflammatory preclinical data
🎯 Whether you’re tracking BTK inhibitors, base editing platforms, or molecular AML therapies, this week’s recap delivers the most impactful advancements in hematology.
📢 Stay Ahead in Hematology Research!
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