As recently reported in CheckOrphan, a new drug for sickle cell disease has quickly ascended through the FDA approval processes in an accelerated review.
A New Sickle Cell Drug
Shrouded in high expectations, the drug producer Novartis believes the drug will be a best seller. The drug ‘crizanlizumab’ is an anti-P-selectin antibod which prevents blood cells from clumping together and creating blockages. These blockages are the cause of vaso-occlusive crises (VOCs), one of the most frequent and unpleasant effects of the disease. VOC’s are potentially fatal but unfortunately common, with the majority of those with the disease in the US visiting the hospital multiple times a year as a result. Such a common medical complication is an expensive cost to the health care system, at an estimated 1.1 billion dollars per year despite only affecting around 100,000 Americans.
Promising Results
The new drug has shown very promising results in the latest trials, with one dose of the drug every month significantly reducing the number of VOC’s that the patients suffered, the number of health care visits, and the number of days hospitalized. These results have been extremely exciting for the medical and affected communities, with the drug promising the potential to alter the daily life by minimizing one of the most tiresome symptoms of those effected.
The medical community has a few potentially exciting leads for treating sickle cell disease, with diverse avenues being examined. Global Blood Therapeutics is also in late-stage development of a drug that helps prevent the sickling and destruction of red blood cells and others who are more farsighted see great potential in gene therapy, with new technologies like CRISPR thought to potentially cure the disease and not just the symptoms.
For now, drugs like crizanlizumab are very promising. The FDA has taken this into account and the drug is receiving a priority review, with supporters hoping it could receive approval within the next few months. If the drug is approved, it is expected to enable people with sickle cell diseases to ease one of the more limiting aspects of the disease.
