Advances in pharmacotherapy for sickle cell disease: what is the current state of play?

Despite over 100 years of neglect and insufficient funding, sickle cell disease has risen to the top of the discussions due to the recent approval of two new genetic therapies. Prior to these approvals, there were only four prior approved medications for sickle cell disease in spite of being the most common inherited blood disorder. The advent and expense of these new genetic therapies have finally brought the trials and tribulations associated with SCD including the suffering and early mortality of affected individuals to the much-needed limelight. Presently, questions about how these therapies will be used and what that means for ongoing pharmaceutical development remain.

Here, we wish to highlight the current medications and treatments for SCD using already published literature as well as scrutinize the tedious process of implementation for these newly approved commercial genetic therapies.

In our expert opinion, despite the progress we have made, significant challenges remain and the most important requirement for any of these treatments is ensuring all affected individuals have access to a sickle cell specialist who can provide comprehensive care.