This month, two Israeli pharmaceutical companies received good news from the US Food and Drug Administration (FDA).

Jerusalem’s Gamida Cell was granted orphan product development status for NiCord, its enriched stem-cell treatment for acute lymphoblastic leukemia, acute myeloid leukemia, Hodgkin lymphoma and myelodysplastic syndrome. Orphan drug designation promotes the development of promising products for the treatment of rare diseases or conditions.

Misgav-based cCAM Biotherapeutics got the green light to proceed with a Phase 1 trial for CM-24, a first-in-class immunomodulatory monoclonal antibody for treating various types of cancers.

Gamida Cell develops cell-therapy technologies and products for transplantation and adaptive immune therapy. The company’s pipeline of products in development address conditions including blood cancers, non-malignant hematological diseases such as sickle-cell disease and thalassemia, neutropenia and acute radiation syndrome, autoimmune diseases, solid tumors and genetic metabolic diseases.

“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 Dr. Yael Margolin.

Two days after the FDA announcement, Gamida Cell revealed that the first person has been successfully transplanted with cryopreserved (frozen) NiCord in the company’s ongoing Phase I/II clinical study for blood-cancer patients. Until now, NiCord has been transplanted as a “fresh” product that must be infused into the patient within a limited number of hours from the moment the product is released from the manufacturing site. (via Israel21c)

[Photo: ששון גואטה / YouTube]