News|Articles|April 1, 2026
FDA Extends Review Timeline of Orca-T for MDS, Leukemia
Author(s)Tony Berberabe, MPH
Fact checked by: Sabrina Serani
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Key Takeaways
- A 3-month PDUFA extension to July 6, 2026 resulted from FDA classification of updated CMC content as a Major Amendment, without new clinical data requests.
- Orca-T comprises purified hematopoietic stem cells, regulatory T cells, and conventional T cells from HLA-matched donors to reduce GVHD while preserving anti-tumor immunity.
The FDA has extended the deadline to review Orca-T for the treatment of myelodsyplastic syndromes and acute leukemias with a PDUFA date of July 6, 2026.
The FDA has extended the review timeline of its Biologics License Application (BLA) for Orca-T for the treatment of patients with myelodysplastic syndromes (MDS) and acute leukemias, according to the manufacturer.1 Orca-T, an investigational allogeneic T-cell immunotherapy derived from matched donors, is designed to reduce graft-versus-host disease while preserving anti-cancer immune effects. The new Prescription Drug User Fee Act (PDUFA) target action date is July 6, 2026.
The extension follows Orca Bio's recent submission of updated chemistry, manufacturing and controls (CMC) information in response to requests from the FDA as part of the standard review process. The FDA classified the submission as a Major Amendment (MA) to the BLA, which does not reflect concerns about safety and efficacy, but does extend the review by 3 months. According to the manufacturer, the FDA has not requested any additional clinical data. Orca Bio believes that the updated information submitted in the amendment does not affect the benefit-risk conclusions of the BLA.
"We appreciate the frequent engagement with the FDA throughout the review process,” Nate Ferhoff, co-founder and chief executive officer at Orca Bio, said. “Our continued focus is on preparing for the potential approval and commercial launch of Orca-T. We remain committed to working with the Agency, physicians and the broader blood cancer community to deliver this important therapy to patients with hematologic malignancies as quickly as possible.”
The BLA for Orca-T was granted
According to a post hoc analysis presented at the
Patients who had undergone reduced-intensity condition were administered Orca-T and demonstrated durable outcomes with a trend toward decreased incidence of acute graft-vs-host disease (aGVHD), acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML), or MDS. Pooled results from patients treated with Orca-T in clinical trials were compared with a historical control cohort from the Center for International Blood and Marrow Transplant Research registry. The control patients had received a conventional allogeneic transplant with posttransplant cyclophosphamide, or PTCy, which is a widely used standard for GVHD prophylaxis.
In a focused analysis of patients aged 18 to 65 years with MDS receiving a myeloablative, HLA-matched transplant, the differences were pronounced. As presented at TANDEM, the Orca-T cohort of 25 patients showed a 1-year overall survival of 100%. This compares with 80% in the 95-patient PTCy cohort. The 2- and 3-year overall survival rates were also 100% for Orca-T vs 70% and 62%, respectively, for PTCy. At 1 year, relapse-free survival was 95% with Orca-T vs 64% with PTCy, and the nonrelapse mortality rate was 0% vs 9.9%.
About Orca-T
Orca-T is composed of highly purified hematopoietic stem cells, regulatory T-cells and conventional T-cells derived from either related or unrelated matched donors. Orca-T has received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation for the prevention of graft versus host disease or death in patients eligible for hematopoietic stem cell transplant from the FDA.
The company has indicated that commercial-scale manufacturing processes are established and that it remains on track for a potential mid-2026 launch, pending approval.
REFERENCES
1. Orca Bio Announces FDA Review Extension of BLA for Orca-T for the Treatment of Hematologic Malignancies. News release. April 1, 2026. https://tinyurl.com/5n98xv5u
2. Orca Bio announces FDA acceptance and priority review of the biologics license application (BLA) for Orca-T to treat hematological malignancies. News release. Orca Bio. October 6, 2025. Accessed April 1, 2026. https://tinyurl.com/mpkppnky
3. Orca Bio presents new data at the 2026 Tandem Meetings of ASTCT and CIBMTR reinforcing Orca-T as a durable, high-precision cell therapy for hematological malignancies. News release. February 5, 2026. Accessed February 9, 2026. https://tinyurl.com/y79r33w4
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