Orca Bio enters commercial market after FDA approval for Tregzi

USA—Orca Bio has entered the commercial cell therapy market after securing U.S. Food and Drug Administration (FDA) approval for Tregzi, the first regulatory T cell-based therapy designed for adults with blood cancers undergoing stem cell transplantation.

The approval marks the company’s first commercial product and introduces a new treatment option that aims to reduce chronic graft-versus-host disease (GVHD), one of the most serious complications of donor stem cell transplants.

A new approach to stem cell transplantation

Tregzi, also known as Orca-T, is an engineered allogeneic cell therapy used alongside matched-donor hematopoietic stem cell transplantation.

Unlike conventional transplants, which infuse a mixed population of donor stem and immune cells, Tregzi separates and enriches specific cell types before infusion.

It contains purified regulatory T cells that suppress harmful immune reactions, hematopoietic stem and progenitor cells that rebuild the blood and immune system, and conventional T cells that help eliminate remaining cancer cells and protect against infections.

Orca Bio Chief Executive Officer Nate Fernhoff said the therapy offers patients a better balance between curing blood cancers and reducing life-threatening transplant complications.

Strong clinical trial results

The FDA based its decision on data from the Phase III Precision-T trial involving 187 adults with acute myeloid leukaemia, acute lymphoblastic leukaemia, myelodysplastic syndrome and mixed-phenotype acute leukaemia.

Participants received either Tregzi with tacrolimus or a standard donor transplant with tacrolimus and methotrexate.

After one year, 78% of patients treated with Tregzi were alive without chronic GVHD, compared with 38% of those receiving standard transplants.

Moderate-to-severe chronic GVHD occurred in 13% of Tregzi recipients versus 44% in the control group, while overall survival reached 94% compared with 83% for conventional transplantation.

The study also reported lower non-relapse mortality and fewer severe infections among patients receiving Tregzi.

Manufacturing and commercial rollout

The approval followed a three-month extension of the review after the FDA requested additional manufacturing information.

According to Fernhoff, close collaboration with regulators helped address the agency’s questions on the complex production process.

Orca Bio currently manufactures Tregzi at its 100,000-square-foot facility in Sacramento, California.

The company aims to deliver each personalised therapy from donor cell collection to hospital infusion within 72 hours.

It has produced more than 500 clinical products and expects manufacturing failure rates to remain in the low single digits because the therapy relies on healthy donor cells rather than patients’ own immune cells.

The company plans to launch Tregzi at a limited number of treatment centres before expanding to about 25 centres by the end of the year.

It also recently announced a new manufacturing facility in Princeton, New Jersey, to increase production capacity and shorten delivery times across the United States.

Pricing and future plans

Orca Bio has set Tregzi’s wholesale acquisition cost at US$428,000.

The company said the price reflects the therapy’s potential to reduce the long-term medical and economic burden associated with chronic GVHD.

It added that reimbursement pathways are already in place through commercial insurers and government programmes to improve patient access.

Earlier this year, Orca Bio raised US$250 million through two financing rounds to support Tregzi’s commercial launch.

Although the biotechnology initial public offering market has seen renewed activity in recent months, Fernhoff said the company remains focused on expanding Tregzi’s availability before pursuing further fundraising opportunities.