FDA Grants Orphan Drug Status to CTD402 for Rare T-Cell Blood Cancers

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Imviva Biotech's CTD402 to accelerate the development of this allogeneic anti-CD7 CAR-T cell therapy for patients with relapsed or refractory T-cell acute lymphoblastic leukemia and lymphoblastic lymphoma (T-ALL/LBL), Imvia announced in a news release.

This regulatory milestone for the clinical-stage biotechnology company highlights an urgent need for new therapeutic options for individuals facing these specific hematologic conditions. Orphan drug designation is a status reserved for therapies intended to treat rare diseases affecting fewer than 200,000 people in the United States. By receiving this designation, the investigational therapy gains access to several development incentives, including tax credits for clinical research, waivers for prescription drug user fees and a period of extended market exclusivity upon potential approval. The designation is particularly significant for patients with rapidly progressing diseases where treatment timing is a critical factor in clinical outcomes.

Main data that support the findings

Early data from the evaluation of CTD402 demonstrate its potential to serve as an effective intervention for patients with cancer who have not responded to previous treatments or have seen their disease return. Clinical findings show that the therapy achieved a 64.1% complete remission (CR) rate among participants. Furthermore, 91.7% of patients reached a status of minimal residual disease (MRD)-negative, meaning that highly sensitive testing could not detect remaining cancer cells in the body. These statistics support the potential of CTD402 to address a critical unmet need in the T-ALL/LBL community.

A key feature of CTD402 is its "off-the-shelf" allogeneic platform. Unlike autologous CAR-T therapies, which require collecting a patient’s own cells and sending them to a laboratory for a manufacturing process that typically lasts one to two months, CTD402 is ready at the point of care. The manufacturing process has shown high levels of consistency across 18 donors and 13 production lots. This immediate availability aims to eliminate life-threatening delays for patients with rapidly progressive disease who cannot afford to wait weeks for personalized cell production.

TENACITY-01 trial details

The therapy is currently being studied in a global clinical trial known as TENACITY-01.This phase 1b/2 trial is a single-arm, open-label study, meaning all participants receive the investigational treatment rather than a placebo and both the researchers and patients are aware of the treatment being administered. The trial is enrolling approximately 54 adolescents and adults aged 12 years and older across sites in the United States, the European Union and the Asia-Pacific region.

The primary goals of the TENACITY-01 trial are to evaluate the safety, efficacy and cellular pharmacokinetics — or how the therapy moves through and is processed by the body — of CTD402. The first patient in the United States was successfully dosed in December 2025. Following the current phase 1b portion, interim data are expected to be available by mid-2026. These results will guide the progression into the phase 2 evaluation. The study is projected to reach completion by late 2028. This schedule is designed to support an accelerated development pathway, which is essential for a patient population facing high mortality rates and limited time for intervention.

Safety of the treatment

Safety is a primary focus of the ongoing TENACITY-01 trial as researchers monitor how the "ready-at-point of care" cells interact with the immune systems of patients with cancer. Because CTD402 is an allogeneic therapy — meaning it uses donor cells rather than the patient's own — it incorporates specific genetic modifications to improve safety and compatibility. The product candidate features a T-cell receptor (TCR) and HLA class 2 knockout. These modifications are intended to reduce the risk of complications associated with donor cells.

Additionally, CTD402 utilizes a proprietary technology known as ANSWER inhibitory ligands. These are designed to enhance the therapy's resistance to host immune rejection, potentially allowing the donor CAR-T cells to persist longer in the patient's system without being attacked by the patient's own immune cells. The FDA has also granted the therapy regenerative medicine advanced therapy designation and rare pediatric disease designation for the treatment of relapsed or refractory T-ALL, further signaling the importance of the safety and efficacy data being gathered in the current clinical settings.

Editor's note: This article is for informational purposes only and is not a substitute for professional medical advice, as your own experience will be unique. Use this article to guide discussions with your oncologist. Content was generated with AI and reviewed by a human editor.

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