French biopharmaceutical firm Erytech Pharma has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for its lead product GRASPA/ERY-ASP for treatment of acute myeloid leukaemia (AML), an aggressive form of blood cancer.
Annually, around 34,000 mainly adults and older patients are affected by AML, the most common form of acute leukemia, in the US and Europe.
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ERY-ASP is called as GRASPA in Europe and is a new enzyme formulation of L-asparaginase, with a safer and broader range of clinical use compared with the current forms due to the entrapment and protection of the enzyme inside homologous red blood cells.
ERY-ASP is currently in Phase III clinical trial for acute lymphoblastic leukaemia (ALL) and in Phase IIb trial for AML in Europe.
The company said that a Phase I trial in adults with acute lymphoblastic leukaemia in the US and also a Phase II trial in pancreas cancer in Europe are being started.
Erytech co-founder and chief scientific officer Yann Godfrin said: “We have been encouraged by the results from our work in acute lymphoblastic leukemia, and are committed to further clinical evaluation of the product in other indications of high unmet need.
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By GlobalData“Acute myeloid leukemia and solid-tumour cancers are investigated to develop a safe and effective therapy, providing a superior treatment outcome. Orphan drug status provides certain benefits that can support us in achieving this goal.”
According to the company, the rationale for using L-asparaginase in acute myeloid leukemia is strong.
As the median of age of AML patients is 70 years, a large majority are too fragile to tolerate the current asparaginase formulations.
The company said that the new orphan drug designation from the US FDA is the seventh ODD for Erytech.
ERY-ASP is currently benefited from ODD in all three of its lead indications, including ALL, AML and pancreas cancer, both in Europe and the US.
Image: Bone marrow aspirate showing acute myeloid leukaemia. Photo: courtesy of VashiDonsk.
