Everything you Need to Know About the New Treatment for Sickle Cell

The FDA approved a new drug to treat sickle cell anemia, giving hope to the 100,000 Americans diagnosed with the lifelong disease. This drug is the first new treatment that has been approved for sickle cell disease in the past 20 years.

The drug, crizanlizumab, is produced by Novartis and is clinically proven to reduce the occurrence of pain crisis episodes experienced by those diagnosed with sickle cell disease.

What is sickle cell anemia?

Sickle cell anemia is a lifelong genetic blood disease that affects about 100,000 Americans, with a majority being Black and African Americans. Globally, millions of people are estimated to have sickle cell disease, and diagnosis continues to rise with 300,000 babies born each year with the condition, primarily in Africa and India.

The disease is debilitating and can result in life-threatening complications such as stroke, organ failure, early death and painful episodes, called pain crises. People diagnosed often experience severe pain, stigma, ER discrimination, depression and high medical debt due to related health complications’ ongoing medical and hospital treatments.

The only established cure for sickle cell is through a bone marrow transplant (also called stem cell transplant). However, this method is only pursued in severe cases because of limited donors, difficulty finding organ matches, and high risk of death, infection and adverse immune response from the transplant procedure. Currently, the focus is on treatment, primarily hydroxyurea therapy and blood transfusions, to manage the disease and prevent complications, pain crises, and death.

Crizanlizumab is clinically proven to reduce the frequency of pain crises

Crizanlizumab is administered by injection into a vein. It was approved by the FDA as a safe and effective treatment for sickle cell disease based on a 52-week (approximately one year) randomized controlled clinical trial with a sample size of 198 patients. Compared to a placebo (inactive treatment), crizanlizumab reduced the frequency of crises by 45%. The drug also reduced the annual average days spent in hospitals to 4 days compared to the 6.87 days experienced on the placebo.

The reduction of hospital emergency room visits and pain crises might reduce the quality of life burdens experienced by people diagnosed with sickle cell anemia. However, due to the small sample size and limited study length, real-world data will continue to be collected to monitor its larger-scale effectiveness, cost-effectiveness and side effects.