Orca Bio’s BLA for Hematologic Malignancy Cell Therapy Orca-T Accepted by FDA With Priority Review

Orca Bio’s biologics license application (BLA) for Orca-T, an investigational allogeneic cell therapy productintended to treat various hematologic malignancies including acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplastic syndromes (MDS), has been accepted by the FDA with priority review.¹

The Prescription Drug User Fee Act (PDUFA) target action date for the BLA has been set at April 6, 2026. The BLA is supported by data from an open-label, randomized, phase 3 clinical trial (Precision-T; NCT05316701). The pivotal, multicenter Precision-T study is evaluating Orca-T against conventional allogeneic hematopoietic stem cell transplant (alloHSCT) for the treatment of AML, ALL, and MDS. Notably, patients treated with Orca-T showed a statistically significant improvement in survival free of moderate-to-severe chronic graft versus host disease(cGvHD) compared to patients treated with alloHSCT, and as such Precision-T met its primary end point.

"A stem cell transplant has been the only potentially curative option for many people with AML, ALL, or MDS, however treatment-related toxicities too often hinder patient recovery,” Nate Fernhoff, PhD, the cofounder and chief executive officer at Orca Bio, said in a statement.¹ “Acceptance of the Orca-T BLA marks a pivotal moment in our ability to deliver a first-in-class therapy designed to improve survival free from complications like GvHD. Supported by positive phase 3 clinical data, today’s regulatory milestone reflects important recognition of the transformative potential of Orca-T. We look forward to working collaboratively with the FDA on the review of our application with the goal of advancing Orca-T and making it available to patients in need.”

Orca-T is intended to serve as an alternative to standard allo-HCT and consists of CD34+ stem cells, regulatory T-cells, and conventional T-cells from the peripheral blood of matched donors.² Unlike in standard allo-HCT, the main component cell types of Orca-T are administered to patients in separate sequential steps, thus letting the regulatory T-cells to migrate to the solid organs first, allowing for the establishment of an immune barrier prior to the arrival of the conventional T-cells.

CGTLive® has previously spoken to Caspian Oliai, MD, MS, the medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center, about results he presented from the earlier phase 1b portion of Precision-T (NCT04013685) at the 2024 Tandem Meetings |Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held in San Antonio, Texas, February 21-24, 2024. Oliai discussed some of the promising results seen up to that point and the future plans for the therapy.

“Based on these promising phase 1B results, Orca-T has the potential to be a reduced-toxicity alternative to conventional allo-transplant,” Oliai told CGTLive.

CGTLive has also spoken to Rawan Faramand, MD, an assistant professor at Moffit Cancer Center, who presented a subgroup analysis of patients with AML who were treated in the phase 1b portion of Precision-T at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting, held May 31 to June 4, in Chicago, Illinois.³ Faramand went over the promising results, discussed challenges in the study, and expressed enthusiasm for the phase 3 portion.

“These new findings presented at ASCO show that Orca-T has the potential to offer curative therapy for patients with AML with very low rates—and in this case 0%—treatment-related mortality...” Faramand told CGTLive. “In doing so, we're keeping the balance of graft versus leukemia—meaning not seeing higher rates of relapse—while still decreasing the risk of toxicities that we typically observe in a standard of care stem cell transplantation. This approach hopefully has the potential to expand curative treatment to many more patients.”

REFERENCES
1. 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 October 7, 2025.https://orcabio.com/orca-bio-announces-fda-acceptance-and-priority-review-of-the-biologics-license-application-bla-for-orca-t-to-treat-hematological-malignancies/
2. Olliai CO, Pantin J, Hoeg RT, et al. Optimizing outcomes with myeloablative conditioning in older patients: efficacy and safety of precision engineered Orca-T in patients > 55 years old with hematologic malignancies. Presented at: 2024 Tandem Meetings, February 21-24, San Antonio, Texas. Abstract #64
3. Pantin JM, Patel SS, FaramandR , et al. Treatment of acute myeloid leukemia with Orca-T. Presented at: 2024 (ASCO) Annual Meeting; May 31-June 4; Chicago, Illinois.