Gamida Cell said today that it won breakthrough therapy designation from the FDA for its lead product candidate, NiCord.
The Jerusalem-based company’s expanded cell graft is designed to be an alternative to bone marrow transplantation for patients with high-risk hematological cancers. Gamida Cell said it plans to launch an international Phase III registration study of the graft before the end of 2016.
Preliminary clinical evaluation of NiCord showed significant improvement over cord blood transplantation, with fewer infections, reduced length of hospitalization and quicker platelet engraftment, the company reported.
Breakthrough therapy designation is reserved for treatments indicated for serious conditions and that have shown to improve at least 1 clinical endpoint over available therapies. Gamida Cell said it hopes that with guidance from the FDA, they can accelerate the review process and get NiCord to patients quickly.
“We are very pleased the FDA has recognized the potential of NiCord to address the unmet clinical need in bone marrow transplantation,” president & CEO Yael Margolin said in prepared remarks. “The breakthrough therapy designation creates the foundation for a joint and concerted effort between the FDA and Gamida Cell to bring this important therapy faster to patients. We look forward to continuing our close cooperation with the FDA and other regulatory agencies to a positive conclusion as we prepare for commercialization.”