FDA grants orphan drug status to new treatment for agressive blood cancers
Over 60% of patients in early trial of CTD402 saw all cancer signs disappear
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The U.S. Food and Drug Administration (FDA) has granted orphan drug status to an experimental treatment from Imviva Biotech that’s being tested in people with relapsing/refractory T-cell acute lymphoblastic leukemia and lymphoblastic lymphoma, two aggressive forms of blood cancer, in a clinical trial.
The FDA awarded this designation, which aims to speed the development of treatments for rare diseases — defined in the U.S. as conditions affecting fewer than 200,000 people — to CTD402, a CAR T-cell therapy. The benefits of such status include reduced regulatory submission fees and tax credits, as well as seven years of market exclusivity if the therapy is ultimately approved.
“Receiving orphan drug designation for CTD402 is an important milestone for patients with [these relapsing or treatment-resistant blood cancers], who urgently need more effective and accessible treatment options,” Jan Davidson-Moncada, MD, PhD, Imviva’s chief medical officer, said in a company press release.
CTD402 is being evaluated in the global Phase 1b/2 TENACITY-01 (NCT07070219) trial, which is enrolling an estimated 54 patients at sites across the U.S. The first participant was dosed at the end of last year.
Early data showed that 64.1% of patients achieved a complete response, meaning the disappearance of all signs of detectable cancer, and 91.7% achieved minimal residual disease, meaning no cancer cells were detected, the company noted.
Still-enrolling TENACITY-01 trial is testing treatment’s effectiveness
T-cell acute lymphoblastic leukemia, known as T-ALL, and lymphoblastic lymphoma, or LBL, are both marked by the uncontrolled growth of immature T-cells, a type of immune cell. ALL is mainly characterized by blood and bone marrow involvement, while LBL primarily manifests as solid tumor masses in immune organs, such as lymph nodes and the thymus. With LBL, there’s typically minimal bone marrow involvement.
CTD402 is an allogeneic CAR T-cell therapy, meaning it’s made from immune T-cells from healthy donors. These cells are engineered to express a chimeric antigen receptor (CAR) that specifically recognizes CD7, a protein found on the surface of T-cells. The modified T-cells, which are more effective at locating and destroying the targeted cancer cells, are then infused into the patient.
The treatment candidate also incorporates other components to enhance resistance to host immune rejection. Unlike autologous therapies that use a patient’s own T-cells, offering lower rejection but slower manufacturing, CTD402 provides an off-the-shelf availability for immediate treatment, eliminating manufacturing delays that can be life-threatening for patients with rapidly progressive disease.
This recognition … [supports] our belief that a truly off‑the‑shelf CAR T therapy, available at the point of care, has the potential to change the treatment paradigm for these rapidly progressing diseases.
According to the company, CTD402’s manufacturing process has demonstrated product consistency across 18 donors and 13 production lots.
The therapy candidate is now being tested in TENACITY-01, a clinical trial involving adults and adolescents, 12 and older, with treatment-resistant or recurrent T-ALL/LBL. It’s an open-label study, meaning that both researchers and participants know the therapy being given.
The trial, which is evaluating the safety and effectiveness of CTD402, is enrolling participants who experienced a relapse after two or more lines of treatment, with the first relapse occurring within one year after the first disease remission.
The main goals of the study are to assess the treatment’s safety and the proportion of patients achieving a complete response after six months. The trial will also evaluate the treatment’s cellular pharmacokinetics, that is, how many T-cells multiply and how long they remain in a patient’s blood.
The FDA has previously granted rare pediatric disease designation and regenerative medicine advanced therapy designation to CTD402 for the treatment of relapsed or refractory T-ALL. These designations aim to accelerate the development and approval of treatments for rare diseases that primarily affect children, as well as cell therapies addressing unmet needs in serious diseases.
The company is pleased with the new orphan drug designation.
“This recognition … [supports] our belief that a truly off‑the‑shelf CAR T therapy, available at the point of care, has the potential to change the treatment paradigm for these rapidly progressing diseases,” Davidson-Moncada said.
