Sickle Cell Disease: Encouraging New Treatments

Sickle Cell Disease: Encouraging New Treatments


The U.S. Food and Drug Administration is
working with patients and stakeholders including researchers and drug companies
to help deliver new treatments to people living with sickle cell disease. Sickle
cell disease is a chronic genetic disorder. It can cause pain, organ damage,
and even stroke. Until recently, patients only had one FDA-approved drug treatment
option in 2017. The FDA approved the first new treatment in nearly 20 years
to reduce acute complications, but treatments, which also can include pain
medication and chronic blood transfusions, are still limited. That’s
why the FDA is encouraging innovation. Now, a growing number of new products are
in the pipeline. Today, companies developing new treatments can ask the
FDA to grant a fast track designation or breakthrough therapy designation to
speed development and review. Because sickle cell disease affects fewer than
200,000 people in the United States each year, the FDA also can give orphan status
to certain products. This gives incentives to developers. The FDA is
committed to helping patients and considers the development of new
innovative treatments a priority.


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