FDA Extends Review Period for Orca-T in Hematologic Malignancies

The FDA has extended its review period for a biologics license application (BLA) seeking approval for Orca-T as a treatment for patients with different hematologic malignancies, according to a press release from the developer, Orca Bio. The agency has changed the Prescription Drug User Fee Act (PDUFA) date to July 6, 2026, for this indication.

Extension of the review period follows the developer submitting new chemistry, manufacturing, and controls data to the FDA as part of the standard review process. The agency has designated this submission as a major amendment to the BLA, necessitating an additional 3 months of review.

The FDA did not request any additional clinical findings associated with Orca-T. According to the developer, the updated information submitted in the amendment should not impact the benefit/risk profile of Orca-T in the BLA.

“We appreciate the frequent engagement with the FDA throughout the review process. Our continued focus is on preparing for the potential approval and commercial launch of Orca-T,” Nate Fernhoff, co-founder and chief executive officer at Orca Bio, stated in the press release.¹ " 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.”

Developers engineered Orca-T as an allogenic T-cell immunotherapy made of highly purified hematopoietic stem cells, regulatory T cells, and conventional T cells derived from related or unrelated matched donors. The agent has previously received regenerative medicine advanced therapy designation and orphan drug designation from the FDA as a therapy for preventing graft-versus-host disease (GVHD) or death among patients who are eligible to undergo hematopoietic stem cell transplant (HSCT).

The FDA granted priority review to Orca-T as a treatment for patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplastic syndrome (MDS) in October 2025.² Supporting data for the BLA came from the phase 3 Precision-T trial (NCT04013685) evaluating the safety, efficacy, and tolerability of Orca-T vs conventional HSCT strategies across these patient populations.

Previous data from the trial showed a 1-year GVHD-free survival rate of 78% (95% CI, 65%-87%) with Orca-T therapy vs 38% (95% CI, 26%-51%) using allogenic HSCT alone (HR, 0.26; P <.00001).³ The estimated overall survival (OS) rate at 1 year was 94% (95% CI, 86%-97%) and 83% (95% CI, 73%-90%) in each respective arm (HR, 0.49; P = .11823). The cumulative incidence of moderate-to-severe chronic GVHD was 13% (95% CI, 5%-23%) vs 44% (95% CI, 31%-56%) in each arm (HR, 0.19; P <.00002).

No new safety signals were identified with Orca-T in the Precision-T trial. Grade 4 or higher infections were reported in 6% of patients in the Orca-T arm and 10% of the allogenic HSCT arm.

As part of the multi-center, open-label Precision-T trial, 187 patients across the US were assigned to receive Orca-T following a myeloablative conditioning regimen or standard-of-care allogeneic HSCT.⁴

The trial’s primary end point was the 12-month event-free rate for moderate or severe chronic GVHD based on end point adjudication committee assessment. Secondary end points included OS, GVHD-free survival, and relapse-free survival at 12 months.

Patients 18 to 65 years old with acute myeloid, lymphoid, or mixed phenotype leukemia in complete remission (CR) or CR with incomplete hematologic recovery with or without known minimal residual disease (MRD) or MDS indicated for allogeneic HSCT and/or therapy-related or secondary MDS with at least 10% blast burden in the bone marrow were eligible for enrollment on the trial.

References

1. Orca Bio announces FDA review extension of BLA for Orca-T for the treatment of hematologic malignancies. News release. Orca Bio. April 1, 2026. Accessed April 2, 2026. https://tinyurl.com/5atyjxbz
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 2, 2026. https://tinyurl.com/urwb7244
3. Orca Bio announces positive results from the pivotal phase 3 study of investigational Orca-T compared to allogeneic stem cell transplant for the treatment of hematologic malignancies. News release. Orca Bio. March 17, 2025. Accessed April 2, 2026. https://tinyurl.com/26edwyup
4. Precision-T: a randomized study of Orca-T in recipients undergoing allogeneic transplantation for hematologic malignancies (Orca-T). ClinicalTrials.gov. Updated March 4, 2026. Accessed April 2, 2026. https://tinyurl.com/y9ubftdy