Sickle cell disease is a group of inherited red blood cell red blood disorders affecting hemoglobin, the protein that carries oxygen through the body.
Sickle cell disease is caused by a mutation in the hemoglobin-beta gene.
Healthy red blood cells with normal hemoglobin (hemoglobin-A) are disc shaped and glide smoothly through the blood vessels. In people with sickle cell disease, the red blood cells contain abnormal hemoglobin (hemoglobin-S) which causes them to become stiff and "sickle" shaped. The sickle cells accumulate in the blood vessels causing blockages, pain, and damage to organs and tissue.
Symptoms of sickle cell disease include anemia resulting from the rapid destruction of the sickle cells in the body, periodic episodes of extreme pain (pain crises), swelling of the hands and feet, frequent infections, delayed growth or puberty, and vision problems. Complications include stroke, acute chest syndrome, pulmonary hypertension, organ damage, splenic sequestration, blindness, leg ulcers, gallstones, priapism, deep vein thrombosis, and high blood pressure and blood clots during pregnancy.
Until recently, the only possible cure for sickle cell disease was a bone marrow transplant.
In December 2023, the U.S. Food and Drug Administration (FDA) approved two new gene therapies for the treatment of sickle cell disease - Casgevy (exagamglogene autotemcel) and Lyfgenia (lovotibeglogene autotemcel).
Casgevy is a CRISPR/Cas9 genome-edited cell therapy and Lyfgenia is an autologous hematopoietic stem cell-based gene therapy. Both gene therapies are a single course of treatment intended to be a one-time fix for the treatment of sickle cell disease in patients 12 years of age or older.
Drugs used to treat Sickle Cell Disease
The medications listed below are related to or used in the treatment of this condition.
For ratings, users were asked how effective they found the medicine while considering positive/adverse effects and ease of use (1 = not effective, 10 = most effective).
Activity
Activity is based on recent site visitor activity relative to other medications in the list.
Rx
Prescription only.
OTC
Over-the-counter.
Rx/OTC
Prescription or Over-the-counter.
Off-label
This medication may not be approved by the FDA for the treatment of this condition.
EUA
An Emergency Use Authorization (EUA) allows the FDA to authorize unapproved medical products or unapproved uses of approved medical products to be used in a declared public health emergency when there are no adequate, approved, and available alternatives.
Expanded Access
Expanded Access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Pregnancy Category
A
Adequate and well-controlled studies have failed to demonstrate a risk to the fetus in the first trimester of pregnancy (and there is no evidence of risk in later trimesters).
B
Animal reproduction studies have failed to demonstrate a risk to the fetus and there are no adequate and well-controlled studies in pregnant women.
C
Animal reproduction studies have shown an adverse effect on the fetus and there are no adequate and well-controlled studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
D
There is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience or studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
X
Studies in animals or humans have demonstrated fetal abnormalities and/or there is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience, and the risks involved in use in pregnant women clearly outweigh potential benefits.
N
FDA has not classified the drug.
Controlled Substances Act (CSA) Schedule
M
The drug has multiple schedules. The schedule may depend on the exact dosage form or strength of the medication.
U
CSA Schedule is unknown.
N
Is not subject to the Controlled Substances Act.
1
Has a high potential for abuse. Has no currently accepted medical use in treatment in the United States. There is a lack of accepted safety for use under medical supervision.
2
Has a high potential for abuse. Has a currently accepted medical use in treatment in the United States or a currently accepted medical use with severe restrictions. Abuse may lead to severe psychological or physical dependence.
3
Has a potential for abuse less than those in schedules 1 and 2. Has a currently accepted medical use in treatment in the United States. Abuse may lead to moderate or low physical dependence or high psychological dependence.
4
Has a low potential for abuse relative to those in schedule 3. It has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 3.
5
Has a low potential for abuse relative to those in schedule 4. Has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 4.
Alcohol
X
Interacts with Alcohol.
Further information
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