This week’s hematology news covers key regulatory milestones, access decisions, late-stage trial progress, and emerging technologies shaping the hematology treatment landscape.
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In Today’s Newsletter
🧪 Ropeginterferon alfa-2b SURPASS-ET published in Lancet Haematology [1] [US • 24 Nov 2025]
Context: Multicentre, open-label, randomized Phase 3 (NCT04285086). Reported fewer thromboembolic events and JAK2 V617F allele-burden reduction.
Key point: In HU-resistant/intolerant ET with leukocytosis, ropeginterferon alfa-2b achieved 43% durable response at months 9–12 vs 6% for anagrelide, per modified ELN criteria.
Implication: May influence prescriber choice and payer reviews pending full data.
🧬 Merck’s ASH 2025 hematology slate, including MK-1045 and bomedemstat [2] [US • 24 Nov 2025]
Context: >20 abstracts at ASH 6–9 Dec; includes waveLINE ADC zilovertamab vedotin updates.
Key point: First presentations for MK-1045 (CD19xCD3) in R/R B-ALL and Phase 2 bomedemstat in PV; nemtabrutinib program updates across CLL/SLL and indolent lymphomas.
Implication: Signals pipeline investment and modality expansion.
🧰 KOMZIFTI (ziftomenib) added to NCCN AML guidelines (Cat 2A) [3] [US • 25 Nov 2025]
Context: KOMET-001 supported approval (CR/CRh 21.4%, median duration 5 months).
Key point: NCCN lists ziftomenib as Category 2A for adult R/R AML with NPM1 mutation following 13 Nov 2025 FDA approval.
Implication: May influence prescriber choice and payer reviews pending full data.
🧱 Sonrotoclax receives FDA Priority Review in R/R mantle cell lymphoma [4] [US • 26 Nov 2025]
Context: Single-arm Phase 1/2 BGB-11417-201 met ORR by IRC; additional efficacy endpoints favorable (details not specified).
Key point: NDA accepted with Priority Review for adults with R/R MCL after BTK inhibitor.
Implication: Introduces competition that may affect pricing and formulary access.
🧫 IASO Bio’s BCMA CAR-T Fucaso approved in Hong Kong for R/R MM [5] [Hong Kong • 27 Nov 2025]
https://www.iasobio.com/info.php?id=298
Context: First China-developed CAR-T approved in Hong Kong; follows NMPA China 2023 and Macau 2025 approvals.
Key point: Hong Kong DoH BLA approval for adults with R/R multiple myeloma after ≥3 prior lines, including a PI and IMiD.
Implication: Introduces competition that may affect pricing and formulary access.
👶 Early Hemlibra in severe hem A infants lowers bleeds (RWE) [6] [28 Nov 2025]
https://hemophilianewstoday.com/news/early-hemlibra-use-prevents-bleeding-babies-hem-a/
Context: Comparative observational analysis summarized; inhibitor development noted in minimally treated subgroup.
Key point: Real-world cohorts suggest early emicizumab prophylaxis is safe and reduces bleeding; previously untreated infants had fewer bleeds than minimally treated.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
🇬🇧 AUCATZYL (obe-cel) recommended by NICE for adult R/R B-ALL via NHS commissioning [7] [UK • 25 Nov 2025]
Context: Based on FELIX study; MHRA conditional MA April 2025.
Key point: NICE draft guidance recommends obe-cel for adults ≥26 years with R/R B-ALL through routine commissioning in England and Wales.
Implication: May expand screening, initiation, and follow-up at scale.
🇪🇺 EC expands Breyanzi to R/R mantle cell lymphoma [8] [EU • 24 Nov 2025]
Context: 41.2% in response at 24 months; safety consistent with known profile.
Key point: EU approval for liso-cel in adult R/R MCL after ≥2 systemic lines including BTK inhibitor; ORR 82.7% and CR 71.6% in TRANSCEND NHL 001.
Implication: May influence prescriber choice and payer reviews pending full data.
🤖 CytoDiffusion AI shows high-sensitivity leukemia cell detection and calibrated uncertainty [9] [26 Nov 2025]
https://scitechdaily.com/ai-blood-cell-analyzer-outperforms-human-experts-in-detecting-leukemia/
Context: Trained on >500k smear images; can flag rare cells and generate realistic synthetics.
Key point: Generative AI model reportedly surpasses human accuracy on morphology and quantifies uncertainty; published in Nature Machine Intelligence.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
Why it matters
- Interferon-based disease-modifying strategies in ET are advancing beyond cytoreduction.
- Multiple CD19 and BCMA cell-therapy access decisions are widening options across EU, UK, and Hong Kong.
- Priority Review for a BCL2 inhibitor in R/R MCL underscores post-BTK unmet need.
- Early pediatric prophylaxis strategies and AI diagnostics may shift workflows and resource allocation.
- ASH presentations signal continued diversification of bispecifics, ADCs, and targeted inhibitors.
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FAQ
What did SURPASS-ET show for ropeginterferon alfa-2b in ET?
Durable response at 9–12 months was 43% vs 6% for anagrelide, with additional hematologic, symptom, and molecular benefits reported; safety was favorable relative to anagrelide [1]. May affect prescriber decisions pending full data review.
What is the regulatory status of sonrotoclax in MCL?
The FDA accepted the NDA and granted Priority Review for adults with R/R MCL after BTK inhibition, supported by a single-arm Phase 1/2 study meeting ORR by IRC [4].
Is KOMZIFTI now in guidelines?
Yes. NCCN added ziftomenib as Category 2A for adult R/R AML with NPM1 mutation following its FDA approval on 13 Nov 2025. The approval cites KOMET-001 (CR/CRh 21.4%, median 5 months) [3].
What did NICE decide on AUCATZYL?
NICE draft guidance recommends obe-cel for adult R/R B-ALL via routine NHS commissioning in England and Wales, based on FELIX and existing conditional approvals [7].
What changed for Breyanzi in Europe?
The EC approved liso-cel for adult R/R MCL after ≥2 lines including a BTK inhibitor, with TRANSCEND NHL 001 showing ORR 82.7% and CR 71.6%; 41.2% remained in response at 24 months per EU label update [8].
How strong is the evidence for early Hemlibra in infants?
Observational cohorts suggest reduced bleeding with early emicizumab prophylaxis, with inhibitor development noted mainly in minimally treated infants; as RWE, interpretation depends on design and confounding control [6].
Entities / Keywords
Ropeginterferon alfa-2b (BESREMi; PharmaEssentia) • Essential thrombocythemia • Anagrelide • JAK2 V617F • MK-1045 (CD19xCD3) • Bomedemstat (LSD1 inhibitor) • Nemtabrutinib (non-covalent BTK) • Zilovertamab vedotin (ROR1 ADC) • KOMZIFTI (ziftomenib; menin inhibitor) • NCCN AML • Sonrotoclax (BCL2 inhibitor) • Mantle cell lymphoma • Equecabtagene autoleucel (Fucaso; IASO Bio; BCMA CAR-T) • AUCATZYL (obecabtagene autoleucel; obe-cel; Autolus) • Breyanzi (lisocabtagene maraleucel; liso-cel) • Emicizumab (Hemlibra) • CytoDiffusion • ASH 2025
References