“We are very encouraged that the EMA has widened NiCord’s orphan drug designation to include BMT, and believe it is an important recognition of the unmet needs in these indications,” said Gamida Cell president and chief executive officer Dr. Yael Margolin. “This broadened regulatory designation adds a key layer of market exclusivity for NiCord as we move forward with our phase 3 study and prepare for commercialization.”
The EMA grants an orphan drug designation to promote the development of products that demonstrate promise of significant benefit for the treatment of rare diseases. Products receiving orphan drug designation are eligible to receive various regulatory and economic benefits, including 10 years of market exclusivity in the EU.
Gamida Cell was previously granted orphan drug designation for NiCord by the EMA for acute myeloid leukemia (AML), and by the US Food and Drug Administration (FDA) for the treatment of several hematologic malignancies including AML, acute lymphoblastic leukemia (ALL), Hodgkin’s lymphoma, myelodysplastic syndromes (MDS) and chronic myelogenous leukemia (CML). Gamida Cell also received FDA Breakthrough Therapy Designation for NiCord in blood cancers.
NiCord is currently being studied in an international, multi-center, phase 3 registration study as a graft for bone marrow transplantation for patients with blood cancer who do not have a rapidly available fully matched donor. The Company announced last month that the first patient in the study had been transplanted.
Gamida Cell is a world leader in cellular and immune therapies for the treatment of cancer and orphan genetic diseases. The company’s pipeline of products are in development to treat a wide range of conditions including cancer, genetic hematological diseases such as sickle cell disease and thalassemia, bone marrow failure syndromes such as aplastic anemia, genetic metabolic diseases and refractory autoimmune diseases. Gamida Cell’s current shareholders include Novartis, Elbit Imaging, Clal Biotechnology Industries, Israel Healthcare Venture, Teva Pharmaceutical Industries, Denali Ventures and Auriga Ventures.
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