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  6. FDA approves allogeneic regulatory T cell-based immunotherapy with HSPC and T cells-vldq for use in matched donor hematopoietic stem cell transplantation for adults with hematologic malignancies
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FDA approves allogeneic regulatory T cell-based immunotherapy with HSPC and T cells-vldq for use in matched donor hematopoietic stem cell transplantation for adults with hematologic malignancies

On June 30, 2026, the Food and Drug Administration approved allogeneic regulatory T cell-based immunotherapy with hematopoietic stem and progenitor cell (HSPC) and T cells-vldq (Tregzi, Orca Bio) for use in matched donor hematopoietic stem cell transplantation (HSCT) with a myeloablative preparative regimen, for hematopoietic and immunologic reconstitution and to improve chronic graft-versus-host disease (cGHVD)-free survival, in the treatment of adults with hematological malignancies.

Full prescribing information for Tregzi will be posted on 2026 Biological License Application Approvals.

Efficacy and Safety

Efficacy was evaluated in Precision-T (NCT05316701), a multicenter, open-label, randomized, controlled trial in adults with acute leukemias or myelodysplastic syndrome (MDS). In total, 187 patients were randomized to receive either Tregzi (n=93) followed by single-agent graft-versus-host disease (GVHD) prophylaxis with tacrolimus or unmanipulated allograft (n=94) followed by GVHD prophylaxis with tacrolimus and methotrexate.

The primary efficacy outcome measure was cGVHD-free survival (cGFS), defined as the time from HSCT to death by any cause or moderate-to-severe cGVHD, as graded per National Institutes of Health consensus criteria and determined by a blinded, independent endpoint adjudication committee. The median cGFS was not estimable (NE) (95% CI: NE, NE) in Tregzi arm versus 7.3 (95% CI: 6.3, 15.5) months in control arm (HR = 0.26 [95% CI: 0.14, 0.47]; P < .00001) after a median follow up of 8.48 (range: 0 to 22) months and 9.03 (range: 0 to 20) months, respectively. The cumulative incidence of moderate-to-severe cGVHD estimate at 12 months was 12.6% (95% CI: 5.3%, 23.1%) for the Tregzi arm and 44.0% (95% CI: 31.3%, 56.1%) for the unmanipulated allograft control arm (HR = 0.19 [95% CI: 0.08, 0.43]; P = .00002). All 88 patients (100%) treated with TREGZI achieved a neutrophil count of 500/mm3 within 28 days of infusion. Of these, 53 patients (60.2%) had neutrophil counts above 500/mm3 on three consecutive days, confirming neutrophil recovery within 28 days of infusion.

The most common adverse reactions (incidence ≥20%) were mucositis, diarrhea, rash, viral infections, infections-pathogen unspecified, abdominal pain, vomiting, nausea, bacterial infections, hemorrhage, acute GVHD, edema, and fungal infections. 
The prescribing information includes warnings and precautions for graft failure, GVHD, infusion reactions, secondary malignancies and malignancies of donor origin, and transmission of infectious agents.

Recommended Dosage

Tregzi consists of three product components that are sequentially administered: HSPCs, regulatory T cells (Tregs), and conventional T cells (Tcons). The targeted doses for each component are as follows: HSPC: ≥1.0 × 106 viable cells/kg and Tregs: 1.3 × 106 to 3.5 × 106 viable cells/kg administered intravenously (IV) on day 0 followed by Tcons: 1.3 × 106 to 6.9 x 106  viable cells/kg administered IV on day +2 to day +3.

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.

This application was granted priority review. Tregzi received orphan drug designation and regenerative medicine advanced therapy designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics. A description of FDA expedited programs for regenerative medicine advanced therapies is in the Guidance for Industry: Expedited Programs for Regenerative Medicine Therapies for Serious Conditions | FDA.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.

For assistance with single-patient INDs for investigational oncology products regulated by the Center for Biologics Evaluation and Research, healthcare professionals may email OTPRPMS@fda.hhs.gov.

Follow the Oncology Center of Excellence on X @FDAOncology. 

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