FDA Grants Fast Track Designation to Dilanubicel for Allogeneic Cord Blood Transplant Patients

The US Food and Drug Administration (FDA) had granted a fast track designation to Nohla Therapeutics’ dilanubicel (NLA101) for patients with high-risk hematologic malignancies receiving an allogeneic cord blood transplant, which is most often used to treat patients with rare cancers like leukemias, lymphomas, multiple myeloma, myelodysplastic syndrome, and other bone marrow disorders.

“FDA Fast Track designation represents another important regulatory milestone for dilanubicel as we continue to pursue accelerated development strategies in the US, Europe and Japan,” said Katie Fanning, president and CEO of Nohla Therapeutics, in a recent statement. “This designation by the FDA further reinforces the significance of dilanubicel’s clinical results to date and its potential as an important treatment option for patients undergoing hematopoietic stem cell transplantation.”

Dilanubicel is a universal donor, off-the-shelf, ex vivo expanded hematopoietic stem and progenitor cell product intended to deliver rapid, transient hematopoiesis. It does not require Human Leukocyte Antigen (HLA) tissue matching—unlike autologous or patient-specific allogeneic cell therapies—and it may also induce long-term immunologic benefits with the potential for improved survival since it was developed to provide short-term, temporary bone marrow function until a patient’s immune system recovers.

A phase 2 single arm study that evaluated the efficacy and safety of dilanubicel in patients with hematologic malignancies who underwent a myeloablative cord blood transplant served as the basis for the designation. When compared with a separate control group, results showed that infusion of dilanubicel was safe and led to faster neutrophil and platelet recovery with improved long-term survival. Additionally, neither severe acute graft-versus-host disease (GVHD) nor transplant-related mortality was reported in the patients treated with dilanubicel.

Currently, dilanubicel is under evaluation in 2 phase 2 trials. One phase 2b randomized trial is set to assess the use of dilanubicel in patients with acute biphenotypic leukemia, acute lymphoblastic leukemia in remission, acute myeloid leukemia (AML) in remission, chronic myelogenous leukemia, and myelodysplastic syndrome. A total of 160 patients with hematologic malignancies undergoing a myeloablative allogeneic cord blood transplant with or without dilanubicel have been enrolled. The primary outcome measure for the trial will be the time to engraftment (ANC greater than or equal to 500) in both arms (standard myeloablative CBT with and without off-the-shelf expanded cord blood progenitors) as measured in the timeframe of 2 years.

The global phase 2 LAUNCH trial is also enrolling AML patients at risk for neutropenia after administration of intensive chemotherapy treatment. Recurrent event rate of Grade 3 or higher bacterial or fungal infection as measured from randomization through follow-up of 84 days post randomization, 30 days post last infusion of NLA101, or 30 days post last infusion of chemotherapy for Control Arm, (whichever is later) will serve as the primary outcome measure.

“We look forward to working with the FDA and other regulatory agencies after we receive top-line results from our randomized Phase 2b study in this patient population,” added Fanning.

Previously, dilanubicel received priority medicine (PRIME) designation from the European Medicines Agency, and an orphan drug designation from the FDA and European Commission.