The Food and Drug Administration (FDA) has granted Orphan Drug Designation to dilanubicel (Nohla Therapeutics) for the reduction of morbidity and mortality associated with hematopoietic stem cell transplant (HSCT). 

Dilanubicel is a universal donor, off-the-shelf-, ex vivo expanded hematopoietic stem and progenitor cell investigational product that provides rapid, transient hematopoiesis while also inducing long-term immunologic benefits. The FDA designation was supported by data from a Phase 2 single arm study in patients with hematologic malignancies who underwent a myeloablative cord blood transplant. Results showed that treatment with dilanubicel led to faster neutrophil and platelet recovery compared to a control group. In addition, infusion of dilanubicel was found to be safe with no severe acute Graft-versus-Host Disease or transplant-related mortality in the dilanubicel-treated group.

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“Dilanubicel has shown encouraging initial activity as a novel cell therapy in patients with hematologic malignancies receiving a cord blood transplant,” said Katie Fanning, President and CEO of Nohla Therapeutics. “We believe the addition of dilanubicel has the potential to make a meaningful difference for these patients and we look forward to having the top-line results from the fully enrolled randomized Phase 2b trial later this year.”

The Company is also investigating dilanubicel in acute myeloid leukemia patients with chemotherapy-induced myelosuppression; the Phase 2 LAUNCH trial is currently enrolling patients.

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