Beta thalassemia is a rare, inherited blood disorder characterized by the reduced production of hemoglobin in the body, leading to a shortage of mature red blood cells and resulting in anemia, a condition that develops when the body does not receive enough oxygen-rich blood.
Beta thalassemia is caused by a defect in the hemoglobin beta (HBB) gene. The HBB gene provides instructions for making a protein called beta-globin, which is a component of the larger protein called hemoglobin that carries oxygen in the red blood cells.
Common symptoms of anemia include weakness, paleness, tiredness, and shortness of breath. Severe anemia can be debilitating and it can lead to further complications and serious health issues if left untreated.
Treatment for anemia associated with beta thalassemia consists mainly of red blood cell transfusions.
Reblozyl (luspatercept-aamt) is FDA-approved for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell transfusions. Reblozyl works as an erythroid maturation agent to regulate late-stage red blood cell maturation, which helps to reduce the red blood cell transfusion burden.
Zynteglo (betibeglogene autotemcel) is an FDA-approved gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions. Zynteglo works by adding functional copies of a modified form of the beta-globin gene into a patient’s own blood stem cells to enable the production of a modified functional hemoglobin, eliminating the need for regular red blood cell transfusions.
Drugs used to treat Beta Thalassemia
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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