FDA Grants Nohla Therapeutics’ Cell Therapy Dilanubicel Orphan Drug Designation for HSCT

Source: Darko Stojanovic, no changes made, CC0 Creative Commons.
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Nohla Therapeutics, headquartered in Seattle, is a leading developer of “off-the shelf” cell therapies for patients with cancer and other diseases. Nohla’s proprietary notch ligand technology platform serves as the foundation for its ongoing clinical, preclinical and discovery programs. The company’s lead product candidate, dilanubicel, is currently under evaluation in two ongoing Phase 2 clinical trials. Nohla is also pursuing multiple preclinical and discovery programs in the areas of immune tolerance and other diseases.

Dilanubicel is a universal donor, off-the-shelf, ex vivo expanded hematopoietic stem and progenitor cell investigational product intended to provide rapid, transient hematopoiesis with long-term immunologic benefits. Unlike autologous or other patient-specific cell therapies, dilanubicel does not require Human Leukocyte Antigen (HLA) tissue matching.

Intentionally developed to provide short-term, temporary bone marrow function until a patient’s immune system recovers, dilanubicel may also induce long-term immunologic benefits with the potential for improved survival. Dilanubicel is manufactured ahead of time, cryopreserved, and intended for immediate off-the-shelf use.

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.

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The U.S. Food and Drug Administration (FDA) has granted Nohla’s lead product candidate, dilanubicel (NLA101), Orphan Drug Designation for reduction of morbidity and mortality associated with hematopoietic stem cell transplant (HSCT). Dilanubicel received Orphan Drug Designation from the European Commission in January 2018.

“Dilanubicel has shown encouraging initial activity as a novel cell therapy in patients with hematologic malignancies receiving a cord blood transplant,” … “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.” Katie Fanning, President and CEO of Nohla Therapeutics.

 

Source:

  1. https://globenewswire.com/news-release/2018/07/16/1537836/0/en/Nohla-Therapeutics-Receives-FDA-Orphan-Drug-Designation-for-Dilanubicel-for-Hematopoietic-Stem-Cell-Transplant-Patients.html