Hematology Today—March 9, 2026

LucidQuest

4 hours ago

This week’s Hematology update highlights regulatory approvals, priority review progress, rare disease momentum, emerging clinical data, and continued innovation across cell therapy and modeling-driven drug development.
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🩸 TECVAYLI plus DARZALEX FASPRO wins U.S. FDA approval in RRMM [1] [US • 05 Mar 2026]

https://www.jnj.com/media-center/press-releases/johnson-johnson-announces-u-s-fda-approval-of-tecvayli-plus-darzalex-faspro-for-relapsed-refractory-multiple-myeloma-offering-a-potential-new-standard-of-care-as-early-as-second-line
Context: Johnson & Johnson said the approval is for adults with relapsed/refractory multiple myeloma after at least one prior line, including a proteasome inhibitor and an immunomodulatory agent.
Key point: TECVAYLI (teclistamab-cqyv) plus DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) gained FDA approval based on Phase 3 MajesTEC-3 data showing improved progression-free survival and overall survival versus standard regimens [1].
Implication: May influence prescriber choice and payer reviews pending full data.

🧪 AN2 moves oral epetraborole into planned Phase 2 PV study [2] [US • 03 Mar 2026]

https://investor.an2therapeutics.com/news-releases/news-release-details/an2-therapeutics-announces-plans-advance-oral-epetraborole-phase
Context: The planned study is for adults with phlebotomy-dependent polycythemia vera, with an open-label sentinel cohort, dose optimization, randomized placebo-controlled portion, and optional extension.
Key point: AN2 Therapeutics said oral epetraborole will advance into Phase 2 in PV, supported by prior clinical and preclinical observations showing controlled, sustained, dose-dependent hematocrit reductions and tolerability in non-PV settings [2].
Implication: May influence prescriber choice and payer reviews pending full data.

🧬 Rusfertide NDA accepted, Priority Review granted in PV [3] [US • 02 Mar 2026]

https://www.takeda.com/newsroom/newsreleases/2026/nda-rusfertide/
Context: Takeda and Protagonist said the filing is primarily supported by Phase 3 VERIFY and four-year efficacy and safety data from Phase 2 REVIVE and THRIVE.
Key point: The FDA accepted the rusfertide NDA for adults with polycythemia vera and granted Priority Review; the companies said VERIFY met the primary endpoint and four key secondary endpoints, including hematocrit control, reduced phlebotomy, and improved patient-reported outcomes [3].
Implication: May influence prescriber choice and payer reviews pending full data.

🧫 LYMPHIR shows early signal before commercial CAR-T in high-risk DLBCL [4] [US • 04 Mar 2026]

https://citiusonc.com/news/news-details/2026/Citius-Oncology-Announces-Preliminary-Topline-Phase-1-Data-from-Study-of-LYMPHIR-E7777-Dosing-Prior-to-Commercial-CART-Therapy-in-HighRisk-Diffuse-Large-BCell-Lymphoma/default.aspx
Context: Investigator-initiated Phase 1, open-label, dose-escalation study in 14 patients with high-risk relapsed/refractory diffuse large B-cell lymphoma receiving LYMPHIR before commercial CD19 CAR-T.
Key point: Citius Oncology reported preliminary topline data showing 86% overall response rate at one month, including 57% complete response and 29% partial response, with no dose-limiting toxicities observed [4].
Implication: May influence prescriber choice and payer reviews pending full data.

🔬 RELAX study reports HAM-Ven activity in relapsed AML [5] [05 Mar 2026]

https://www.eurekalert.org/news-releases/1118845
Context: Dresden investigators described RELAX as a multicentre, single-arm, phase 1/2 study of venetoclax plus high-dose cytarabine and mitoxantrone (HAM-Ven) in relapsed or refractory acute myeloid leukemia.
Key point: The study team reported remission rates rising from about 40% with conventional chemotherapy to 75% with HAM-Ven, with many patients proceeding to stem cell transplant [5].
Implication: May influence prescriber choice and payer reviews pending full data.

🖥️ Certara says Simcyp PBPK work replaced ten human asciminib studies [6] [US • 03 Mar 2026]

https://www.certara.com/pressrelease/certara-simcyp-simulator-results-replace-ten-human-trials-for-chronic-myeloid-leukemia-cml-therapy-asciminib/
Context: Certara said physiologically-based pharmacokinetic modeling with Simcyp supported the new drug application for asciminib (Scemblix), a BCR::ABL1 allosteric inhibitor for chronic myeloid leukemia.
Key point: The company said FDA accepted PBPK modeling results in lieu of certain clinical pharmacology studies, with the published case study stating at least ten dedicated human trials were replaced [6].
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

🧷 Starton files CAR-T combination patents around STAR-LLD lenalidomide platform [7] [US • 05 Mar 2026]

https://www.startontx.com/starton-therapeutics-files-patent-applications-for-the-use-of-car-t-therapies-in-combination-with-its-proprietary-continuous-low-dose-immunomodulatory-therapy-star-lld-a-formula/
Context: Starton said the patent filings cover use of continuous low-dose lenalidomide formulation STAR-LLD with in vivo and ex vivo CAR-T therapies in blood cancers.
Key point: The company filed patent applications aimed at combination use with CAR-T, arguing continuous low-dose delivery could support T-cell fitness and reduce toxicity versus conventional IMiD exposure [7].
Implication: Signals pipeline investment and modality expansion.

🌍 PYRUKYND approved in the UAE for adult thalassemia [8] [UAE • 02 Mar 2026]

https://investor.agios.com/news-releases/news-release-details/agios-pyrukyndr-mitapivat-approved-adults-thalassemia-united
Context: Agios said the Emirates Drug Establishment approved PYRUKYND (mitapivat) for adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia; NewBridge will continue commercialization in the Gulf region.
Key point: Agios said PYRUKYND is now approved in the UAE for adult thalassemia and is the only medicine approved there for both transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia [8].
Implication: Introduces competition that may affect pricing and formulary access.

🩹 Sutacimig wins FDA Breakthrough Therapy Designation in Glanzmann thrombasthenia [9] [US • 05 Mar 2026]

https://www.hemab.com/news-items/hemab-therapeutics-receives-fda-breakthrough-therapy-designation-for-sutacimig-in-glanzmann-thrombasthenia
Context: Hemab said the designation covers prevention of bleeding episodes in Glanzmann thrombasthenia and is supported by Phase 1/2 multiple ascending dose data.
Key point: The FDA granted Breakthrough Therapy Designation to sutacimig after Hemab reported clinically meaningful reductions in bleeding, including severe events requiring high-intensity treatment [9].
Implication: May influence prescriber choice and payer reviews pending full data.

Why it matters

Multiple hematology categories moved at once this week, including FDA approval, NDA acceptance, Breakthrough designation, and regional approval [1][3][8][9].
Polycythemia vera is becoming a crowded innovation area, with rusfertide nearing an FDA decision and epetraborole entering Phase 2 development [2][3].
Cell-therapy adjacencies remain active, from LYMPHIR as a pre-CAR-T immunomodulatory strategy to Starton’s patent filings around lenalidomide-based CAR-T combinations [4][7].
Academic and model-informed development stories also stood out, with RELAX in AML and Simcyp-enabled PBPK work for asciminib showing non-traditional paths to evidence generation [5][6].
Rare hematology continues to expand geographically and regulatorily, including UAE thalassemia approval for mitapivat and Breakthrough designation for sutacimig in GT [8][9].

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FAQ

What exactly was approved for TECVAYLI and DARZALEX FASPRO?

Johnson & Johnson said the FDA approved TECVAYLI plus DARZALEX FASPRO for adults with relapsed/refractory multiple myeloma who have received at least one prior line, including a proteasome inhibitor and an immunomodulatory agent [1].

How far along is rusfertide in polycythemia vera?

Takeda and Protagonist said the FDA accepted the NDA and granted Priority Review. The filing is mainly based on Phase 3 VERIFY plus long-term Phase 2 REVIVE/THRIVE data, with a PDUFA target in 3Q26 [3].

Is epetraborole already in Phase 2 for PV?

Not yet. AN2 said it plans to initiate the Phase 2 study in 3Q26, subject to regulatory clearance, with potential data updates starting as early as 4Q26 [2].

What did the RELAX study show in AML?

The Dresden-led team reported that venetoclax plus high-dose cytarabine and mitoxantrone increased remission rates to 75% in relapsed/refractory AML in the phase 1/2 study, compared with around 40% cited for conventional chemotherapy [5].

What is notable about the UAE PYRUKYND decision?

Agios said the Emirates Drug Establishment approved PYRUKYND for adult alpha- or beta-thalassemia, whether transfusion-dependent or non-transfusion-dependent, and described it as the only approved medicine there for this population [8].

Why does sutacimig’s Breakthrough designation matter?

Hemab said sutacimig is being developed for prophylaxis in Glanzmann thrombasthenia, a setting with no effective prophylactic options currently available. Breakthrough designation can speed development and review when preliminary evidence suggests substantial improvement [9].

Entities / Keywords

TECVAYLI (teclistamab-cqyv), DARZALEX FASPRO (daratumumab and hyaluronidase-fihj), MajesTEC-3, relapsed/refractory multiple myeloma, RRMM, bispecific antibody, BCMA [1]
Epetraborole, AN2 Therapeutics, polycythemia vera, PV, phlebotomy-dependent PV, hematocrit control [2]
Rusfertide, Takeda, Protagonist Therapeutics, VERIFY, REVIVE, THRIVE, hepcidin mimetic [3]
LYMPHIR (E7777; denileukin diftitox-cxdl), Citius Oncology, DLBCL, CAR-T, regulatory T cells, Tregs [4]
RELAX, HAM-Ven, venetoclax, cytarabine, mitoxantrone, relapsed AML, stem cell transplant [5]
Certara, Simcyp, PBPK, asciminib, Scemblix, chronic myeloid leukemia, CML [6]
Starton Therapeutics, STAR-LLD, lenalidomide, IMiDs, CAR-T combination patents [7]
PYRUKYND (mitapivat), AQVESME, Agios, NewBridge Pharmaceuticals, thalassemia, ENERGIZE, ENERGIZE-T [8]
Sutacimig (HMB-001), Hemab Therapeutics, Glanzmann thrombasthenia, GT, Breakthrough Therapy Designation, prophylaxis [9]