According to a story from PMLive, the US Food and Drug Administration (FDA) has recently begun the priority review process for a new potential therapy from pharmaceutical behemoth Novartis. This experimental drug goes by the name crizanlizumab and the company is convinced that it could be a major game changer in the treatment of sickle cell anemia, a rare illness that significantly shortens lifespan and predominately affects people of African ancestry.
About Sickle Cell Anemia
Sickle cell anemia is a genetic, inherited disorder of the blood. This disorder is characterized by an abnormality affecting hemoglobin, which is found in red blood cells and is responsible for carrying oxygen. The abnormality causes blood cells to lose their typical circular shape and instead become take on an elongated, sickle-like appearance. This is caused by a genetic mutation that may have arisen as a defense against malaria, although this benefit only occurs in people with sickle cell trait, not the disease. Symptoms begin to appear at around six months old and include swelling of the hands and feet, stroke, bacterial infections, and acute episodes of severe pain termed sickle cell crisis. Severity of disease varies, but these attacks can result in serious declines in health and organ damage. Treatment is mostly symptomatic, but bone marrow transplant has been curative in children. Life expectancy is between 40 and 60. To learn more about sickle cell anemia, click here.
Treating Sickle Cell Crisis
Sickle cell crisis is often triggered by the abnormally shaped blood cells sticking together and causing clots. Their distinctive shape increases the likelihood of such clots occurring. Crizanlizumab is an antibody that is tailored to prevent the sickle cells from clumping together and forming these harmful and painful clots. Novartis says that as many as 60 percent of sickle cell anemia patients experience sickle cell crisis at least twice per year.
Encouraging Findings
The accelerated review of the therapy is based on encouraging data from a phase 2 clinical trial in which a monthly treatment with the antibody was found to reduce the chance of crisis. 36 percent of patients using the drug that had suffered at least two crisis episodes per year prior to treatment saw no crises when using crizanlizumab compared to only 18 percent in the placebo control group.
From this evidence, it appears that this antibody has some significant therapeutic potential for sickle cell anemia patients.
