Blood Cancer Treatment Granted Orphan Drug Designation

The FDA today granted orphan drug designation to an experimental drug that treats several types of blood cancers.

Gamida Cell’s NiCord is being developed for the treatment of acute lymphoblastic leukemia, acute myeloid leukemia, Hodgkin lymphoma, and myelodysplastic syndrome. NiCord acts as an expanded cell graft derived from an entire umbilical cord blood unit and enriched with stem cells.

“Receipt of orphan drug status for NiCord in the US and Europe advances Gamida Cell’s commercialization plans a major step further, as both afford significant advantages,” said Gamida Cell president and CEO Yael Margolin in a press release. “We very much appreciate the positive feedback and support of the FDA and EMA and look forward to continuing what has been a very positive dialogue with these important agencies.”

The drug is derived from a single cord blood unit that has been expanded in culture and enriched with stem cells utilizing the manufacturer’s proprietary NAM technology. NiCord is currently being evaluated in clinical trials as a therapeutic treatment for hematological malignancies, in which the drug is being used as the sole stem cell source.

“We have witnessed in the clinic a small glimpse of the potential of NiCord that we have extensively studied in the lab and animal models,” said Gamida Cell co-founder Tony Peled in a press release. “The novel finding of the first NiCord phase I/II study is that cord blood-derived hematopoietic stem cells that are expanded in culture are capable of providing both, fast and robust long-term engraftment. This outstanding clinical outcome inspires us to continue developing our entire pipeline of therapeutic stem cell treatments.”