Hematology Video Recap — October 20, 2025

Join us for this biweekly roundup of Hematology news, where we summarize important announcements, study results, and innovation trends driving the field forward.

Watch Our Video Summary Capturing Top Hematology News from the Last Two Weeks

Top Stories Covered in This Video

🧪 Iopofosine I-131 CMA eligibility at EMA for post-BTKi WM [1] [EU • 06 Oct 2025]

https://www.cellectar.com/news-media/press-releases/detail/376/cellectar-biosciences-announces-european-medicines-agency
Context: SAWP advice following scientific procedure, PRIME in place, CLOVER WaM Phase 2 cited.
Key point: EMA said a CMA filing is eligible for iopofosine I-131 in post-BTKi refractory WM, submission planned early 2026.
Implication: May influence prescriber choice and payer reviews pending full data.

🧬 FDA Priority Review for Orca-T in AML, ALL, MDS [2] [US • 06 Oct 2025]

https://orcabio.com/orca-bio-announces-fda-acceptance-and-priority-review-of-the-biologics-license-application-bla-for-orca-t-to-treat-hematological-malignancies/
Context: Phase 3 Precision-T met primary endpoint of improved survival free of moderate to severe chronic GvHD vs alloHSCT.
Key point: FDA accepted the BLA and granted Priority Review, PDUFA 06 Apr 2026.
Implication: May influence prescriber choice and payer reviews pending full data.

🇨🇦 pCPA LOI opens public reimbursement path for HEMGENIX [3] [Canada • 06 Oct 2025]

https://newsroom.csl.com/2025-10-06-CSL-Behring-Signs-Letter-of-Intent-with-pan-Canadian-Pharmaceutical-Alliance-pCPA-for-Public-Reimbursement-of-HEMGENIX-R-etranacogene-dezaparvovec-,-the-First-Gene-Therapy-for-Hemophilia-B
Context: Health Canada authorization for adults, sponsor cites HOPE-B outcomes and safety profile.
Key point: CSL Behring signed an LOI with pCPA enabling provinces and territories outside Quebec to proceed with listings.
Implication: May expand screening, initiation, and follow-up at scale.

🧬 Orca-T Priority Review, duplicate source item [5] [US • 06 Oct 2025]

https://orcabio.com/orca-bio-announces-fda-acceptance-and-priority-review-of-the-biologics-license-application-bla-for-orca-t-to-treat-hematological-malignancies/
Context: Same Phase 3 Precision-T dataset as item [2].
Key point: Confirms BLA acceptance and Priority Review for Orca-T, PDUFA 06 Apr 2026.
Implication: May influence prescriber choice and payer reviews pending full data.

🧫 Sonrotoclax gets FDA BTD in relapsed or refractory MCL [6] [US • 13 Oct 2025]

https://www.businesswire.com/news/home/20251013125479/en/BeOne-Medicines-Sonrotoclax-Granted-Breakthrough-Therapy-Designation-by-U.S.-FDA
Context: Based on Phase 1/2 BGB-11417-201 in post-BTKi, anti-CD20 treated MCL, Phase 3 CELESTIAL-RRMCL underway.
Key point: FDA granted Breakthrough Therapy Designation and accepted Project Orbis participation.
Implication: May influence prescriber choice and payer reviews pending full data.

🧯 MNV-201 earns FDA Orphan Drug in MDS [7] [US • 15 Oct 2025]

https://finance.yahoo.com/news/minovia-therapeutics-receives-fda-orphan-113000608.html
Context: Mitochondrial cell therapy platform, Fast Track already in MDS, RPDD and Fast Track in Pearson syndrome.
Key point: FDA granted Orphan Drug Designation for MNV-201 in myelodysplastic syndrome.
Implication: Signals pipeline investment and modality expansion.

🧒 KRESLADI BLA resubmission accepted, PDUFA 28 Mar 2026 [8] [US • 14 Oct 2025]

https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-announces-fda-acceptance-bla-resubmission
Context: Global Phase 1/2 showed 100% survival at 12 months post infusion, fewer significant infections, well tolerated.
Key point: FDA accepted BLA resubmission for severe LAD-I; PRV eligibility if approved.
Implication: May influence prescriber choice and payer reviews pending full data.

🤝 Novo Nordisk and Omeros sign zaltenibart deal up to 2.1B dollars [9] [15 Oct 2025]

https://www.globenewswire.com/news-release/2025/10/15/3167091/0/en/Novo-Nordisk-and-Omeros-announce-asset-purchase-and-license-agreement-for-Omeros-clinical-stage-MASP-3-inhibitor-zaltenibart-OMS906.html
Context: Novo gains global rights to MASP-3 inhibitor zaltenibart, Omeros retains certain preclinical MASP-3 rights.
Key point: Asset purchase and license agreement valued up to 2.1B dollars in upfront and near-term milestones.
Implication: Signals pipeline investment and modality expansion.

Why It Matters

Accelerated pathways continue across hematology, with EU CMA eligibility and multiple US Priority Reviews in play.
Canada’s pCPA LOI can translate high-cost gene therapy into provincially funded access.
FDA designations, BTD and ODD, may shorten timelines and inform payer expectations.
Two H1 2026 PDUFAs cluster catalysts for allogeneic cell therapy and rare immunology.
Novo’s complement-pathway move highlights sustained interest in rare hematology and renal targets.

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FAQ

What is Cellectar preparing to file in Europe and when?

A CMA for iopofosine I-131 in post-BTKi refractory Waldenström macroglobulinemia, planned for early 2026 after SAWP advice confirmed filing eligibility [1][4].

What is Orca-T’s US review status?

FDA accepted the BLA and granted Priority Review with a PDUFA date of 06 Apr 2026, supported by Phase 3 Precision-T showing benefit in survival free of moderate to severe chronic GvHD vs alloHSCT [2][5].

Does the pCPA LOI make HEMGENIX publicly reimbursed now?

Not by itself. The LOI enables provinces and territories outside Quebec to proceed with listing decisions. Each jurisdiction must finalize its formulary listing [3].

What did FDA grant for sonrotoclax and why is Project Orbis noted?

Breakthrough Therapy Designation for adult relapsed or refractory MCL, with Orbis participation to support concurrent multi-agency submission and review [6] (endpoint specifics not stated here).

What are the key timelines for Rocket’s KRESLADI?

FDA accepted the resubmitted BLA for severe LAD-I. PDUFA is 28 Mar 2026. Sponsor notes 12-month survival and infection reductions in Phase 1/2 support [8].

What is the scope of the Novo–Omeros agreement?

Novo acquires and licenses zaltenibart with global rights, total potential value up to 2.1B dollars, while Omeros retains certain preclinical MASP-3 rights [9].

Entities / Keywords

Cellectar Biosciences, iopofosine I-131, Waldenström macroglobulinemia, EMA, SAWP, PRIME; Orca Bio, Orca-T, AML, ALL, MDS, alloHSCT, chronic GvHD, Priority Review, PDUFA; CSL Behring, HEMGENIX, hemophilia B, pCPA, Canada, reimbursement; BeOne Medicines, sonrotoclax, BCL2 inhibitor, mantle cell lymphoma, Project Orbis, BTD; Minovia Therapeutics, MNV-201, mitochondrial cell therapy, myelodysplastic syndrome, ODD, Fast Track; Rocket Pharmaceuticals, KRESLADI, marne-cel, LAD-I, PRV; Novo Nordisk, Omeros, zaltenibart, MASP-3, complement pathway, PNH.