FDA grants orphan drug designation to ERYTECH’s lead product for AML treatment

ERYTECH Pharma (Paris:ERYP) (Euronext Paris: FR0011471135 - ERYP), a French biopharmaceutical company that develops innovative treatments for acute leukemia and other oncology indications with unmet medical needs, announces that its lead product GRASPA®/ERY-ASP has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of Acute Myeloid Leukemia (AML), an aggressive form of blood cancer.

Orphan Drug Designation (ODD) is generally granted to drugs or biologics intended for treatment of rare diseases and disorders of high unmet medical need, affecting fewer than 200,000 people in the USA. This designation conveys special incentives to the sponsor, including seven years of US market exclusivity for the drug or biologic upon FDA approval, a prescription drug user fee waiver, and certain tax credits.

AML, the most common form of acute leukemia, affects about 34,000 new patients per year in the US and Europe, mainly adults and older patients, for which few therapeutic options are available today.

ERY-ASP, in Europe known as GRASPA®, is a new enzyme formulation of L-asparaginase, with a safer and broader range of clinical use than existing forms due to the entrapment and protection of the enzyme inside homologous red blood cells. ERY-ASP is in Phase III clinical trial for Acute Lymphoblastic Leukemia (ALL) and in Phase IIb trial for AML in Europe. A Phase I study in adult ALL is being launched in the USA as well as a Phase II study in pancreas cancer in Europe.

The rationale for using L-asparaginase in AML is strong. However, as the median of age of AML patients is about 70 years old, a large majority of them are too fragile to tolerate the current asparaginase formulations. Thanks to its better safety profile, ERY-ASP aims to make asparaginase available also to AML patients.

The new Orphan Drug Designation is the seventh ODD for ERYTECH. The dossier was filed after the positive safety review by an independent Data Safety Monitoring Board (DSMB) of the first 30 patients treated in the ongoing Phase IIb study.

ERY-ASP now benefits from ODD in all three of its lead indications, ALL, AML and pancreas cancer, both in Europe and the USA.

"We are very pleased by the granting of this additional Orphan Drug Designation" said Dr Yann Godfrin, co-founder and Chief Scientific Officer of ERYTECH. "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. Acute Myeloid Leukemia and solid-tumor 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."