Approved treatments for sickle cell disease

Adakveo (crizanlizumab-tmca) is an antibody-based medication approved for use in adults and adolescents, ages 16 and older, who experience vaso-occlusive crises caused by SCD. Given as an into-the-vein or intravenous infusion, Adakveo is designed to reduce how often these painful crises happen.

Casgevy (exagamglogene autotemcel), formerly known as CTX001 or exa-cel, is an approved gene-editing therapy used for people with SCD who experience recurrent vaso-occlusive crises. It is designed to reduce the frequency and severity of these painful crises. The one-time therapy is designed to be administered via an intravenous or into-the-vein infusion.

Endari (L-glutamine) is an oral therapy that’s approved in the U.S. to reduce acute disease complications, such as pain crises, in children and adults with SCD, ages 5 and older. The therapy is available as an oral powder that’s dissolved in a liquid or soft food prior to administration.

Ferriprox (deferiprone) is an oral medication that’s used to treat iron overload that occurs as a complication of blood transfusions in children and adults with SCD. The therapy is supplied in the form of oral tablets and as an oral solution.

Hydroxyurea is an oral medication that’s approved to reduce the frequency of painful crises and lessen the need for blood transfusions in people with sickle cell anemia, the most common and often the most severe form of SCD. There are three brands of hydroxyurea currently available for treating sickle cell in the U.S., Droxia, Siklos, and Xromi, each with its own formulation (oral capsules, tablets, and solution).

Lyfgenia (lovotibeglogene autotemcel), formerly known as LentiGlobin or lovo-cel, is a one-time gene therapy approved for people with SCD with a history of vaso-occlusive events. It is designed to reduce the frequency and severity of these events, including vaso-occlusive crises and acute chest syndrome. The therapy is given via a single intravenous or into-the-vein infusion.

Nonsteroidal anti-inflammatory drugs, or NSAIDs, are a class of anti-inflammatory, fever-reducing medications that are commonly used to help relieve pain in people with SCD. There are many different types of NSAIDs, including common over-the-counter pain relievers, such as ibuprofen and aspirin, as well as others that are only available with a medical prescription.

 Oxbryta (voxelotor) is an oral therapy that’s conditionally approved in the U.S. to prevent red blood cell sickling and destruction in children and adults with SCD, ages 4 and older. The therapy is supplied in the form of oral tablets or tablets for oral suspension.

Opioids are a class of compounds with pain relieving properties (analgesics) that are used for managing moderate to severe pain, including pain associated with SCD. There are several types of naturally-occurring and lab-made (synthetic) opioids that can be used for this purpose, and they can be given in different strengths and through distinct routes of administration based on medical need.