Hematology Video Recap—November 3, 2025

GSK has received US FDA approval for Blenrep, also known as belantamab mafodotin-blmf, in combination with bortezomib and dexamethasone for relapsed or refractory multiple myeloma after at least two prior lines of therapy. The Phase 3 DREAMM-7 trial, which enrolled 494 patients, demonstrated that this combination reduced the risk of death by 51 percent and tripled median progression-free survival to 31.3 months compared with 10.4 months on a daratumumab-based triplet. The approval could significantly shift prescriber and payer preferences once full data become available.

Heidelberg Pharma has announced that its lead antibody-drug conjugate, HDP-101, has received FDA Fast Track designation for multiple myeloma. The ongoing Phase 1/2a trial focuses on relapsed and refractory disease. The designation enables rolling review and more frequent engagement with regulators, accelerating the development of this Amanitin-based BCMA-targeting ADC.

Syndax Pharmaceuticals has secured FDA approval for Revuforj, or revumenib, expanding its indication to include adult and pediatric patients with relapsed or refractory acute myeloid leukemia harboring an NPM1 mutation. This decision, based on data from the Phase 2 AUGMENT-101 study, makes Revuforj the first and only FDA-approved therapy for both NPM1-mutated AML and acute leukemia with KMT2A translocation, reinforcing Syndax’s leadership in precision hematology.

The UK’s Medicines and Healthcare products Regulatory Agency has approved Novo Nordisk’s concizumab, marketed as Alhemo, for prophylaxis in patients aged 12 and older with hemophilia A or B with inhibitors. Approved via the International Recognition Procedure, this once-daily subcutaneous therapy inhibits tissue factor pathway inhibitor, restoring clotting function independently of factor VIII or IX. The approval is expected to support broader adoption and enhance patient management pathways.

Lantern Pharma presented a remarkable complete metabolic response in a patient with diffuse large B-cell lymphoma following CAR-T and bispecific therapies. The finding came from an ongoing Phase 1 trial evaluating LP-284 in relapsed or refractory B-cell lymphomas. After two cycles, the patient achieved complete remission, underscoring LP-284’s potential to address therapy-resistant disease and the growing impact of AI-enabled drug design in oncology.

Moleculin Biotech has highlighted progress in its pivotal Phase 3 MIRACLE study evaluating Annamycin, branded AnnAraC, in combination with cytarabine for relapsed or refractory acute myeloid leukemia. Annamycin, a next-generation anthracycline with non-cardiotoxic properties, holds orphan designations from both the FDA and EMA. Results from this global trial could redefine the therapeutic landscape for high-risk AML patients.

GeneVentiv Therapeutics has received positive FDA INTERACT feedback supporting advancement of its gene therapy candidate, GENV-HEM, for hemophilia A with or without inhibitors. The agency endorsed the company’s development path toward an Investigational New Drug submission. The therapy uses an adeno-associated viral vector platform, marking an important step forward in gene therapy innovation.

BioMarin Pharmaceutical announced plans to divest its hemophilia A gene therapy, Roctavian, following persistently low commercial performance. In the third quarter of 2025, Roctavian generated three million dollars in revenue despite a list price of 2.9 million dollars per treatment. The divestiture will maintain therapy availability in the United States, Germany, and Italy while enabling BioMarin to refocus resources on other portfolio priorities.

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FAQ

What differentiates Blenrep (GSK) from other BCMA-targeting agents?

Blenrep is the only BCMA antibody–drug conjugate approved for community use with a simplified REMS program [1].

What is unique about HDP-101 (Heidelberg Pharma)?

HDP-101 is an Amanitin-based ADC targeting BCMA, designed to deliver a potent RNA polymerase II inhibitor selectively to myeloma cells [2].

How does Revuforj (Syndax) expand AML treatment?

It is now FDA-approved for both NPM1-mutated and KMT2A-rearranged acute leukemias in adults and children [3].

How does concizumab (Novo Nordisk) differ from factor replacement?

It inhibits tissue factor pathway inhibitor, restoring clotting independently of factor VIII or IX [4].

What is the therapeutic rationale of LP-284 (Lantern Pharma)?

LP-284 exploits DNA-repair deficiencies via transcription-coupled nucleotide excision repair, retaining efficacy despite TP53 mutations [5].

Why is BioMarin divesting Roctavian?

Low uptake and high cost led to weak sales; divestiture allows patient continuity while freeing portfolio resources [8].

Entities / Keywords

Blenrep (belantamab mafodotin-blmf); GSK plc; HDP-101 (pamlectabart tismanitin); Heidelberg Pharma; Revuforj (revumenib); Syndax Pharmaceuticals; Concizumab (Alhemo); Novo Nordisk; LP-284; Lantern Pharma; Annamycin (naxtarubicin); Moleculin Biotech; GENV-HEM; GeneVentiv Therapeutics; Roctavian (valoctocogene roxaparvovec); BioMarin Pharmaceutical; FDA; MHRA; EMA.

References

1. https://www.gsk.com/en-gb/media/press-releases/blenrep-approved-by-us-fda-for-use-in-treatment-of-relapsedrefractory-multiple-myeloma/
2. https://heidelberg-pharma.com/en/press-investors/announcements/press-releases-and-ad-hoc-announcements/pr-heidelberg-pharmas-lead-adc-candidate-hdp-101-granted-fast-track-designation-by-us-fda-for-the-treatment-of-multiple-myeloma
3. https://ir.syndax.com/news-releases/news-release-details/syndax-announces-fda-approval-revuforjr-revumenib-adult-and
4. https://www.gov.uk/government/news/concizumab-approved-to-prevent-or-reduce-the-frequency-of-bleeding-episodes-in-people-aged-12-years-and-older-with-haemophilia-a-or-b-with-inhibitors
5. https://ir.lanternpharma.com/news-1/news/news-details/2025/Lantern-Pharma-Presents-LP-284-Clinical-Data-at-25th-LLM-Congress-Highlighting-Complete-Response-in-Therapeutically-Exhausted-DLBCL-Patient–Therapeutic-Potential-in-Advanced-B-Cell-Cancers-/default.aspx
6. https://moleculin.com/moleculin-to-highlight-its-ongoing-phase-3-acute-myeloid-leukemia-miracle-clinical-trial-at-the-14th-annual-acute-leukemia-meeting/
7. https://www.geneventiv.com/2025/10/30/geneventiv-therapeutics-announces-positive-fda-interact-feedback-supporting-advancement-of-genv-hem-gene-therapy-for-hemophilia-a-with-or-without-inhibitors/
8. https://www.fiercepharma.com/pharma/biomarin-resigned-ending-journey-hemophilia-gene-therapy-roctavian-seeking-divestiture-amid