Medicines in development for sickle cell disease in USA

Sickle cell disease (SCD) is the most common inherited blood disorder in the USA, afflicting nearly 100,000 Americans. Patients with SCD have sickle-shaped red blood cells, which have trouble moving through the blood vessels and thus cause blockages or slow blood flow and oxygen to parts of the body.

SCD is life-threatening, due to potential complications from blocked blood vessels, which can include stroke, difficulty breathing, pulmonary hypertension and other organ damage, according to a posting on the website of Pharmaceutical Research and Manufacturers of America by Andrew Powaleny is director of public affairs at PhRMA.

As SCD impacts fewer than 200,000 patients it is considered a rare disease. However, SCD disproportionately affects African Americans, with the disease occurring in nearly 1 in every 365 Black or African American births and 1 in 13 are born with the sickle cell trait. Additionally, the American health system loses nearly $1 billion each year in direct medical costs associated with SCD, and hospitalizations due to its related complications total $488 million annually.