Hematology Today—March 16, 2026

This week’s Hematology update highlights regulatory filings, positive late-stage clinical data in multiple myeloma, mixed AML trial outcomes, rare disease progress, and gene therapy manufacturing expansion.

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💊 TECVAYLI filed at EMA for earlier-line RRMM [1] [EU • 10 Mar 2026]

https://www.jnj.com/media-center/press-releases/tecvayli-teclistamab-monotherapy-application-submitted-to-the-ema-for-relapsed-refractory-multiple-myeloma-after-at-least-one-prior-therapy
Context: Johnson & Johnson said the filing is supported by Phase 3 MajesTEC-9 in 614 patients with relapsed/refractory multiple myeloma after 1–3 prior lines.
Key point: TECVAYLI (teclistamab) monotherapy was submitted to the EMA for adults with relapsed/refractory multiple myeloma after at least one prior therapy, with reported PFS and OS improvement versus PVd or Kd.
Implication: May influence prescriber choice and payer reviews pending full data.

🧬 Mezigdomide posts positive Phase 3 interim in RRMM [2] [09 Mar 2026]

https://news.bms.com/news/corporate-financial/2026/Bristol-Myers-Squibb-Announces-Positive-Phase-3-Results-from-the-SUCCESSOR-2-Study-of-Oral-Mezigdomide-in-Relapsed-or-Refractory-Multiple-Myeloma/default.aspx
Context: SUCCESSOR-2 is a randomized, open-label Phase 2/3 study comparing mezigdomide plus carfilzomib and dexamethasone with carfilzomib and dexamethasone in relapsed/refractory multiple myeloma.
Key point: Bristol Myers Squibb reported statistically significant and clinically meaningful PFS improvement for oral mezigdomide in the MeziKd regimen versus Kd alone; full data will be presented later.
Implication: May influence prescriber choice and payer reviews pending full data.

🩸 SELLAS starts frontline AML Phase 2 for SLS009 [3] [US • 12 Mar 2026]

https://ir.sellaslifesciences.com/news/News-Details/2026/SELLAS-Life-Sciences-Announces-Enrollment-of-First-Patient-in-Newly-Diagnosed-First-Line-AML-Trial-of-SLS009/default.aspx
Context: The randomized Phase 2 trial plans to enroll about 80 newly diagnosed AML patients in the US and Europe, including a predictive biomarker cohort and an early-resistance cohort.
Key point: SELLAS enrolled the first patient in a first-line AML study of SLS009 (tambiciclib), focused on patients unlikely to benefit from azacitidine plus venetoclax or showing lack of response after two cycles.
Implication: May influence prescriber choice and payer reviews pending full data.

⚠️ Gilteritinib misses OS endpoint in newly diagnosed FLT3m+ AML [4] [09 Mar 2026]

https://newsroom.astellas.com/2026-03-09-astellas-and-hovon-confirm-phase-3-study-did-not-meet-its-primary-endpoint-of-overall-survival-in-patients-with-newly-diagnosed-flt3m-aml
Context: The Phase 3 HOVON 156 / AMLSG 28-18 / PASHA study compared XOSPATA (gilteritinib) with midostaurin-based treatment in newly diagnosed FLT3-mutated AML eligible for intensive chemotherapy.
Key point: Astellas and HOVON said the study did not meet its primary endpoint of overall survival at the primary analysis; adverse event rates were described as similar between arms.
Implication: May influence prescriber choice and payer reviews pending full data.

🧪 Ruxoprubart advances in PNH, SC route cleared for study [5] [US • 09 Mar 2026]

https://www.globenewswire.com/news-release/2026/03/09/3251828/0/en/NovelMed-Therapeutics-Announces-Regulatory-Clearance-for-Subcutaneous-Ruxoprubart-and-Reports-Positive-Phase-II-Intravenous-Monotherapy-Results-in-PNH.html
Context: NovelMed described a 12–16-week open-label Phase II IV monotherapy study in treatment-naïve adult PNH and said SC Phase II trials are planned but not yet initiated.
Key point: NovelMed reported positive Phase II IV monotherapy results for ruxoprubart in PNH and said the subcutaneous route has regulatory clearance for further Phase II development.
Implication: May influence prescriber choice and payer reviews pending full data.

📄 Pierre Fabre secures FDA Type A meeting for tabelecleucel BLA [6] [US • 12 Mar 2026]

https://www.prnewswire.com/news-releases/pierre-fabre-pharmaceuticals-announces-type-a-meeting-agreed-with-us-food-and-drug-administration-fda-for-tabelecleucel-biologic-license-application-bla-302711648.html
Context: The meeting follows a Complete Response Letter dated 09 Jan 2026 for tabelecleucel in relapsed/refractory Epstein-Barr virus positive post-transplant lymphoproliferative disease.
Key point: Pierre Fabre Pharmaceuticals said the FDA agreed to a Type A meeting to discuss a path forward for the tabelecleucel BLA and potential accelerated approval strategy.
Implication: Introduces competition that may affect pricing and formulary access.

🏭 Lonza expands ZYNTEGLO manufacturing with Genetix [7] [US • 09 Mar 2026]

https://www.lonza.com/news/2026-03-09-14-00
Context: Lonza said commercial manufacturing will continue at its Houston site, with room for future scale-up for additional Genetix therapies.
Key point: Lonza and Genetix Biotherapeutics extended their commercial manufacturing agreement to expand capacity for ZYNTEGLO (betibeglogene autotemcel), described as the only FDA-approved gene therapy for pediatric and adult transfusion-dependent beta-thalassemia.
Implication: Signals pipeline investment and modality expansion.

Why it matters

Multiple myeloma drove the week’s strongest efficacy headlines, with TECVAYLI moving toward an earlier-line EU filing and mezigdomide adding a positive Phase 3 interim result [1][2].
AML updates were mixed, with SELLAS moving SLS009 into newly diagnosed disease while Astellas and HOVON reported a Phase 3 setback for gilteritinib in frontline FLT3-mutated AML [3][4].
Rare hematology remained active, with NovelMed positioning ruxoprubart around monotherapy potential in PNH and a future self-administered SC route [5].
Regulatory process mattered as much as data, with Pierre Fabre’s Type A meeting creating a near-term watchpoint for tabelecleucel after a CRL [6].
Manufacturing and supply continued to matter in cell and gene therapy, with Lonza and Genetix extending capacity for ZYNTEGLO amid reported demand growth [7].

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FAQ

What changed for TECVAYLI this week?

Johnson & Johnson said it submitted a Type II variation to the EMA to expand TECVAYLI (teclistamab) monotherapy into relapsed/refractory multiple myeloma after at least one prior therapy. The filing is based on MajesTEC-9 Phase 3 data, with full results still pending broader presentation [1].

What did SUCCESSOR-2 show for mezigdomide?

Bristol Myers Squibb reported a positive interim Phase 3 result for oral mezigdomide plus carfilzomib and dexamethasone in relapsed/refractory multiple myeloma. The company said PFS improved versus Kd, while survival follow-up continues (full dataset not yet disclosed) [2].

Why is the SELLAS SLS009 trial notable?

The study moves SLS009 (tambiciclib) into newly diagnosed first-line AML and targets patients predicted to do poorly on azacitidine plus venetoclax, or who show early resistance. It also uses biomarker and AI-assisted patient-selection approaches, according to the company [3].

What happened in the PASHA study with gilteritinib?

Astellas and HOVON said the Phase 3 HOVON 156 / AMLSG 28-18 / PASHA study did not meet its primary endpoint of overall survival in newly diagnosed FLT3-mutated AML. Secondary endpoints and subgroup analyses are still being evaluated [4].

What is the latest on ruxoprubart in PNH?

NovelMed reported positive Phase II IV monotherapy results in treatment-naïve PNH and said the subcutaneous route has regulatory clearance for Phase II study. The SC program has not started yet, based on the company statement [5].

Why does the FDA Type A meeting matter for tabelecleucel?

Pierre Fabre said the meeting will address the BLA after the FDA issued a Complete Response Letter on 09 Jan 2026. The company framed it as a step toward agreeing a resubmission path and potential accelerated approval strategy in relapsed/refractory EBV+ PTLD [6].

Entities / Keywords

TECVAYLI, teclistamab, Johnson & Johnson, MajesTEC-9, relapsed/refractory multiple myeloma, RRMM, BCMA-CD3 bispecific
mezigdomide, Bristol Myers Squibb, SUCCESSOR-2, MeziKd, CELMoD, targeted protein degradation, RRMM
SLS009, tambiciclib, SELLAS Life Sciences, acute myeloid leukemia, AML, azacitidine, venetoclax, CDK9 inhibitor
XOSPATA, gilteritinib, Astellas, HOVON, PASHA, HOVON 156, AMLSG 28-18, FLT3-mutated AML
ruxoprubart, NM8074, NovelMed Therapeutics, paroxysmal nocturnal hemoglobinuria, PNH, Bb, alternative pathway complement
tabelecleucel, Pierre Fabre Pharmaceuticals, FDA Type A meeting, biologics license application, EBV+ PTLD
ZYNTEGLO, betibeglogene autotemcel, Lonza, Genetix Biotherapeutics, transfusion-dependent beta-thalassemia, gene therapy, manufacturing capacity