The US Food and Drug Administration (FDA) has granted orphan designation for eryaspase to treat acute myeloid leukemia (AML).
Eryaspase, also known as ERY-ASP or GRASPA, is L-asparaginase encapsulated in red blood cells.
These donor-derived, enzyme-loaded red blood cells function as bioreactors to eliminate circulating asparagine and “starve”
leukemic cells, thereby inducing their death.
Research has suggested this delivery system provides improved pharmacodynamics. It protects L-aspariginase from circulating proteolytic enzymes and prevents early liver or renal clearance.
The system also appears to reduce the risk of adverse events.
Eryaspase is currently under investigation in a phase 3 trial for acute lymphoblastic leukemia (ALL) and a phase 2b trial for AML in Europe. A phase 1 study in adult ALL is being launched in the US.
Eryaspase now has orphan designation for ALL, AML and pancreatic cancer, both in Europe and the US.
In the US, orphan designation is generally granted for drugs or biologics intended to treat disorders of high unmet medical need that affect fewer than 200,000 people.
This designation conveys special incentives to the product’s sponsor, including 7 years of US market exclusivity for the drug or biologic upon FDA approval, a prescription drug user fee waiver, and certain tax credits.
