Enzon Pharmaceuticals, Inc has announced the US Food and Drug Administration (FDA) completed its review of the company's Investigational New Drug (IND) application for the use of recombinant human mannose-binding lectin (rhMBL) for the prevention and treatment of severe infections in patients with low levels of MBL undergoing liver transplant treatment.
"We believe rhMBL may have utility across a broad range of life-threatening infections and have identified liver transplant patients as our next area of study. Given that MBL is secreted in the liver, there is a strong scientific rationale for its potential use in treating these patients," said Jeffrey H. Buchalter, Enzon's chairman and chief executive officer. "Further, MBL-deficient patients can be readily identified through protein measures and genotyping which may provide an improved opportunity to personalize anti-infection treatment for immunosuppressed patients."
The FDA previously approved an IND for rhMBL for the prevention and treatment of severe infections in patients with multiple myeloma with low levels of MBL undergoing high-dose chemotherapy and haematopoietic stem cell transplantation in August 2006. Both the liver transplant and multiple myeloma trials are expected to begin enrollment later this year.
rhMBL is a protein therapeutic being developed for the prevention and treatment of severe infections in individuals with low levels of Mannose-Binding Lectin (MBL). Over 10 per cent of the general population is estimated to be MBL-deficient. MBL deficiency may explain why some but not all individuals who are immunosuppressed develop infectious complications even when they receive prophylactic anti-infectious treatment. Studies have shown a correlation between low MBL levels and susceptibility to serious infections in patients immunosuppressed from chemotherapy, including patients with multiple myeloma undergoing high-dose chemotherapy and haematopoietic stem cell transplantation.
MBL is a natural human plasma protein that plays an important role in the humoral innate immune defense. It specifically recognizes a broad range of microorganisms, including bacteria, fungi, viruses, and parasites, through common carbohydrate structures located on their surfaces. MBL binds to the microorganisms, resulting in activation of secondary immune effector mechanisms, such as the complement system, leading to enhanced phagocytosis, killing and clearance of the invading microorganism.