From Canadian access decisions in multiple myeloma to next-gen menin inhibitors and expedited pathways in rare heme cancers, this roundup spotlights developments shaping treatment and market access across Canada, the UK, the US, and the EU.
💡 Key Highlights:
💊 Canada’s CDA recommends conditional public reimbursement for belantamab mafodotin combinations BPd and BVd in RRMM, referencing DREAMM-7 and DREAMM-8, with pCPA talks urged next.
🧬 CHARM Therapeutics nominates CHM-029, a next-gen pan-menin inhibitor for AML discovered via AI DragonFold, showing dose-dependent tumor regression in pre-clinical models and activity against known resistance mutations, first-in-human targeted Q2 2026.
📈 Kura Oncology doses the first patient in the second KOMET-017 Phase 3 trial in newly diagnosed NPM1-mutated or KMT2A-rearranged AML across intensive and non-intensive regimens, triggering a 30 million dollar Kyowa Kirin milestone, 105 million dollars received to date.
🧪 FDA grants Orphan Drug Designation to Leukogene’s M2T-CD33, LTI-214, an MHCII-engager targeting CD33-positive blasts and leukemic stem cells in AML.
📄 Otsuka files an EMA marketing application for all-oral Inaqovi, decitabine–cedazuridine, plus venetoclax in newly diagnosed unfit AML, supported by Phase I–II ASCERTAIN-V reports, complete remission 46.5 percent at a median 2.4 months per source.
🧫 March Biosciences secures FDA RMAT designation for MB-105, an autologous CD5-targeted CAR-T in relapsed or refractory CD5-positive T-cell lymphoma, enabling enhanced FDA interactions and potential expedited review.
🩸 Germany approves Prufibry, a fibrinogen concentrate, for congenital and acquired deficiency across ages, with Austria and Spain expected via decentralized procedure, US BLA under review.
🧷 Zydus Lifesciences receives FDA Orphan Drug Designation for desidustat in beta-thalassemia, supporting orphan incentives and potential 7-year exclusivity upon approval.
🎯 Whether you track myeloma access, AML innovation, T-cell lymphoma cell therapies, or coagulation products, this edition delivers the signals that matter for clinical practice and payer strategy.
Chapters:
0:00 Introduction
0:08 Canada: CDA recommends conditional public reimbursement for Blenrep combos BPd and BVd in RRMM
0:40 CHARM Therapeutics nominates CHM-029, next-gen pan-menin inhibitor for AML, first-in-human planned Q2 2026
1:09 Kura Oncology starts dosing in second KOMET-017 Phase 3 for NPM1-m or KMT2A-r AML, triggering 30 million dollar milestone
1:37 FDA grants Orphan Drug Designation to Leukogene’s M2T-CD33, LTI-214, for AML
2:05 Otsuka submits EMA application for all-oral Inaqovi, decitabine–cedazuridine, plus venetoclax in unfit AML
2:36 March Biosciences secures FDA RMAT for MB-105, CD5-targeted CAR-T, in relapsed or refractory T-cell lymphoma
3:10 Germany approves Prufibry fibrinogen concentrate for congenital and acquired deficiency; Austria–Spain next; US BLA pending
3:37 Zydus Lifesciences receives FDA Orphan Drug Designation for desidustat in beta-thalassemia
3:56 How to reach us
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