FDA Grants 'Orphan Drug' Status to Umbilical Cord Blood-Based Treatment

Gamida Cell's experimental drug treatment NiCord will have Orphan Drug Status if it makes it to market.

NiCord is being explored as a treatment for four different subtypes of blood cancers: acute lymphoblastic leukemia, acute myeloid leukemia, Hodgkin's lymphoma, and myelodysplastic syndrome.

"Receipt of orphan drug status for NiCord in the U.S. 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.”

What is NiCord?

Using Gamida Cell's proprietary technology, NiCord is made from a single cord blood unit that has been expanded in culture and enriched with stem cells. In current clinical trials, NiCord 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.”

Source: Pharmacy Times