Hematology biweekly video highlighting regulatory actions across cancer and bleeding disorders, including approvals, label updates, priority reviews, and guideline changes, alongside clinical progress and access developments.
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Top Stories Covered in This Video
Chapters
0:00 Introduction
0:08 FDA Approves Darzalex Faspro D-VRd for transplant-ineligible newly diagnosed multiple myeloma, with higher MRD negativity and PFS benefit in CEPHEUS 0:45 FDA Grants Fast Track to Innovent IBI3003 trispecific T cell engager for heavily pretreated relapsed or refractory multiple myeloma
1:17 Health Canada Approves Jaypirca, pirtobrutinib, for relapsed or refractory mantle cell lymphoma and CLL/SLL
1:53 NCCN Lists ropeginterferon alfa-2b-njft, BESREMi, as Category 1 preferred regimen for high-risk essential thrombocythemia with inadequate or lost response
2:25 FDA Removes Yescarta label limitation in relapsed or refractory primary central nervous system lymphoma
2:51 Bristol Myers Squibb partners with Kasey Keller to share Breyanzi lymphoma journey for World Cancer Day
3:18 US FDA Grants Orphan Drug Designation to Zydus desidustat for sickle cell disease
3:48 FDA Grants Priority Review to HYMPAVZI, marstacimab, for hemophilia A and B with inhibitors and children 6–11 without inhibitors
4:27 How to reach us
Transcript
Welcome to the latest edition of Hematology Updates, covering breakthroughs in the past two weeks. Brought to you by LucidQuest.
First, in the United States, the FDA approved the D-VRd regimen of Darzalex Faspro, daratumumab plus hyaluronidase, added to bortezomib, lenalidomide, and dexamethasone for newly diagnosed multiple myeloma that is transplant ineligible. Approval cites deeper responses with higher minimal residual disease negativity and a progression free survival benefit versus VRd in the Phase 3 CEPHEUS study, overall survival remains immature, so ongoing follow up will matter for payers and prescribers.
Next, Innovent’s IBI3003, a trispecific T cell engager targeting GPRC5D, BCMA, and CD3, received FDA Fast Track for relapsed or refractory multiple myeloma after four or more prior lines. Early ASH data at higher dose levels showed promising overall response rates with a tolerable safety profile, and a Phase 1 or 2 program is ongoing in China and Australia with a United States trial planned.
In Canada, Jaypirca, pirtobrutinib, the first non covalent reversible BTK inhibitor, is now approved and available for adults with relapsed or refractory mantle cell lymphoma and chronic lymphocytic leukemia or small lymphocytic lymphoma. The decision is based on BRUIN Phase 1 or 2 monotherapy cohorts in patients previously treated with a covalent BTK inhibitor with or without a BCL 2 inhibitor, introducing competition that may affect pricing and formulary access.
The NCCN updated guidelines now list ropeginterferon alfa 2b njft, BESREMi, as a Category 1 preferred regimen for high risk essential thrombocythemia with inadequate or lost response to existing therapy. Use in ET remains investigational pending an FDA label expansion, but the Category 1 designation may inform practice decisions and payer discussions.
Turning to cellular therapy, the FDA updated the Yescarta label by removing the prior Limitations of Use in relapsed or refractory primary central nervous system lymphoma. This change is supported by an investigator sponsored Phase 1 study from Dana Farber, with manageable safety and no new signals reported, potentially influencing prescriber choice while full data mature.
On awareness and access, Bristol Myers Squibb partnered with Kasey Keller to share his lymphoma journey with Breyanzi for World Cancer Day. The campaign highlights gaps in education and access for CAR T in non Hodgkin lymphoma, and underscores Breyanzi as a one time personalized therapy with indications across five cancer types, which may streamline initiation through telehealth and logistics.
Zydus received US FDA Orphan Drug Designation for desidustat in sickle cell disease. Desidustat is an oral hypoxia inducible factor prolyl hydroxylase inhibitor, Phase 2 proof of concept in SCD is completed with publication pending, and ODD may provide tax credits, user fee waivers, and seven years of market exclusivity upon approval.
Finally, the FDA granted Priority Review to the HYMPAVZI, marstacimab, supplemental biologics application in hemophilia A and B with inhibitors, and in children 6 to 11 years without inhibitors. The submission is supported by the Phase 3 BASIS and BASIS KIDS studies with a PDUFA action date expected in the second quarter of 2026. If approved, marstacimab would be the first non factor prophylaxis for children 6 to 11 with hemophilia B, and it holds Breakthrough Therapy designation for pediatric hemophilia B.
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Why it matters
- CD38 quadruplet approval in NDMM may shift frontline myeloma standards toward MRD-driven regimens [1].
- Trispecific engagement targeting BCMA and GPRC5D aims to reduce antigen escape in late-line myeloma [2].
- Non-covalent BTKi entry expands post-cBTKi options, potentially prolonging BTK pathway utility [3].
- Guideline recognition can accelerate ET access while FDA review continues [4].
- Label clarity in PCNSL could widen CAR-T eligibility in a high-need population [5].
🗓️ Explore weekly details and sources
📚 See the full Hematology archive on our research hub page.
FAQ
What exactly did FDA approve for Darzalex Faspro on 27 Jan 2026?
D-VRd for newly diagnosed multiple myeloma patients ineligible for ASCT, supported by CEPHEUS showing deeper responses and PFS improvement versus VRd; OS not mature [1].
How far along is Innovent’s IBI3003 program?
It has FDA Fast Track for heavily pretreated R/R MM, with Phase 1/2 ongoing in China and Australia and a US Phase 1/2 planned; early data suggest promising activity and manageable safety (endpoint details vary by cohort) [2].
Who in Canada can receive Jaypirca now?
Adults with R/R MCL after ≥2 lines including a BTKi, or CLL/SLL after ≥2 lines including a BTKi and a BCL-2 inhibitor, under NOC/c based on BRUIN response data [3].
Is ropeginterferon approved in ET after the NCCN update?
Not yet in the US. NCCN lists it as Category 1 preferred for high-risk ET with inadequate response, while the FDA sBLA for label expansion is under review [4].
Did Yescarta gain a new indication for PCNSL?
The FDA removed the prior Limitations of Use for R/R primary CNS lymphoma, citing manageable safety; clinicians now have clearer labeling for eligible patients (efficacy endpoints not detailed here) [5].
What would HYMPAVZI’s Priority Review change if approved?
It would extend use to patients 6+ with inhibitors and to children 6–11 without inhibitors, including the first non-factor prophylaxis for pediatric hemophilia B in that age group [8].
Entities / Keywords
Darzalex Faspro (daratumumab and hyaluronidase); D-VRd; CEPHEUS; Johnson & Johnson • IBI3003; GPRC5D/BCMA/CD3; Innovent • Jaypirca (pirtobrutinib); Eli Lilly; BRUIN; non-covalent BTK inhibitor • Ropeginterferon alfa-2b-njft (BESREMi); PharmaEssentia; NCCN; ET • Yescarta (axicabtagene ciloleucel); Kite; Gilead; PCNSL • Breyanzi (lisocabtagene maraleucel); BMS; DTC awareness • Desidustat; Zydus; HIF-PHI; SCD; ODD • HYMPAVZI (marstacimab); Pfizer; TFPI; BASIS; Priority Review.
References
https://en.innoventbio.com/InvestorsAndMedia/PressReleaseDetail?key=577