Hematopoietic and stromal cell differentiation.
image by Terese Winslow (assisted by Lydia Kibiuk)
The U.S. Food and Drug Administration (FDA) 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.
Under the Orphan Drug Act of 1983, the FDA provides incentives for companies developing treatments that are expected to provide significant therapeutic advantage over existing treatments, and that target rare medical conditions affecting fewer than 200,000 U.S. patients per year. Incentives include seven-year market exclusivity, tax credits on U.S. clinical trials, fast-tracking of regulatory proceedings, exemption from certain fees, such as waiver of filing fees under the Prescription Drug User Fee Act (PDUFA), and orphan drug grants.
“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.”