ADA-SCID
Adenosine deaminase severe combined immune deficiency (ADA-SCID) is an ultra-rare, inherited genetic disorder, caused by a deficiency in the adenosine deaminase (ADA) enzyme, that is often fatal if left untreated. The ADA enzyme is most active in lymphocytes, a type of white blood cell that is a key part of the body’s immune system.
ADA-SCID results from defects in the ADA gene, which reduce or eliminate the amount of ADA enzyme activity and increase the risk of severe and recurring infections.
ADA-SCID is an ultra-rare disease. It is estimated to occur in approximately one in 200,000 to one in one million newborns around the world.
ADA-SCID is typically diagnosed within the first few months of life. Diagnosis is made through a simple blood test which measures ADA enzyme activity and/or through genetic testing for the gene mutations in a patient with suspected or proven SCID. Sometimes, SCID/ADA-SCID is diagnosed later in childhood in less severely affected individuals using the same testing as in infants.
Without early diagnosis and effective treatment, babies with ADA-SCID usually die from infections before they reach age 2. Treatment is most successful in infants who have not yet experienced serious infections.
Babies born with more severe genetic defects that lead to SCID do not have sufficient immune function to combat effectively infections from bacteria, viruses, or fungi.
Today, all 50 states, the District of Columbia, and Puerto Rico are screening newborns for SCID. Newborn screening may not find less severe cases of ADA deficiency.
REVCOVI
Revcovi is a new enzyme replacement therapy (ERT) for the treatment of adenosine deaminase deficiency severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.
Revcovi is a PEGylated recombinant adenosine deaminase (rADA) enzyme that has been developed to replace the bovine ADA (nADA) that is used in the manufacture of Adagen® (pegademase bovine injection). PEG is polyethylene glycol, a substance that is used to increase the half-life of a linked molecule. Revcovi is the product of recombinant technology, thus eliminating the need to source the adenosine deaminase (ADA) enzyme from animals.
A recombinant protein is a protein that results from the expression of genetically-engineered DNA molecules within living cells.
The dosing schedule for Revcovi will vary from patient to patient. Your healthcare provider will provide an individualized treatment and schedule.
GLOSSARY
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ADA: Stands for adenosine deaminase, an enzyme that removes buildup in your cells.
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ADA-SCID: Stands for adenosine deaminase severe combined immune deficiency, which is a rare genetic disorder that prevents the body from making enough functioning ADA.
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Enzyme: A protein that performs a specific job. Your body has many enzymes.
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Enzyme replacement therapy: A treatment that replaces a missing enzyme in the body.
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dAXP: Stands for deoxyadenosine nucleotide, which is a toxic buildup that can happen in the cells of people with ADA-SCID. dAXP can harm the immune system by damaging and removing lymphocytes.
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Genetic: Something that can be passed down from parent to child.
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Lymphocyte: An immune cell that helps the body fight infections.
Important Safety Information
Warnings and Precautions:
- If you have lower than normal platelet counts, you may be at increased risk of bleeding at the injection site. Your doctor should not prescribe Revcovi if you have severely low blood platelet counts.
- There may be a delay in improvement of your immune system’s function after starting Revcovi treatment. During this delay, it is important to be protected from exposure to infections until your immune system improves.
What are the possible side effects of Revcovi?
- Cough and vomiting were the most common side effects reported by patients receiving Revcovi.
- Based on reports of side effects of other enzyme replacement therapies used to treat ADA-SCID, which are similar to Revcovi, patients taking Revcovi may also experience:
- Blood and blood cell–related problems: red blood cells being destroyed faster than they can be made, resulting in anemia; and higher or lower than normal number of platelets in the blood
- Skin-related problems: redness and itching at the injection site
- Cancers of the immune system called lymphomas
What types of monitoring will be needed while I am taking Revcovi?
- Continuous therapy and adherence to the recommended drug schedule is important for the success of the treatment.
- While you are being treated with Revcovi, your doctor will need to do blood tests to monitor the levels of ADA activity and metabolites and overall immune function.
- This monitoring will ensure you are maintaining appropriate levels for therapeutic benefit. If an ongoing decline in your blood’s measured adenosine deaminase (ADA) activity occurs, your immune function and clinical status will be monitored closely and precautions will be taken to help reduce your risk of infection.
What is known about taking Revcovi during pregnancy?
- If you are pregnant or planning to become pregnant, or breastfeeding or planning to breastfeed, please talk to your doctor.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.
Indication
Revcovi® (elapegademase-lvlr) is indicated for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.
Please see the Full Prescribing Information.
Important Safety Information
Warnings and Precautions:
- If you have lower than normal platelet counts, you may be at increased risk of bleeding at the injection site. Your doctor should not prescribe Revcovi if you have severely low blood platelet counts.
- There may be a delay in improvement of your immune system’s function after starting Revcovi treatment. During this delay, it is important to be protected from exposure to infections until your immune system improves.
What are the possible side effects of Revcovi?
- Cough and vomiting were the most common side effects reported by patients receiving Revcovi.
- Based on reports of side effects of other enzyme replacement therapies used to treat ADA-SCID, which are similar to Revcovi, patients taking Revcovi may also experience:
- Blood and blood cell–related problems: red blood cells being destroyed faster than they can be made, resulting in anemia; and higher or lower than normal number of platelets in the blood
- Skin-related problems: redness and itching at the injection site
- Cancers of the immune system called lymphomas
What types of monitoring will be needed while I am taking Revcovi?
- Continuous therapy and adherence to the recommended drug schedule is important for the success of the treatment.
- While you are being treated with Revcovi, your doctor will need to do blood tests to monitor the levels of ADA activity and metabolites and overall immune function.
- This monitoring will ensure you are maintaining appropriate levels for therapeutic benefit. If an ongoing decline in your blood’s measured adenosine deaminase (ADA) activity occurs, your immune function and clinical status will be monitored closely and precautions will be taken to help reduce your risk of infection.
What is known about taking Revcovi during pregnancy?
- If you are pregnant or planning to become pregnant, or breastfeeding or planning to breastfeed, please talk to your doctor.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.
Indication
Revcovi® (elapegademase-lvlr) is indicated for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.
Please see the Full Prescribing Information.
References available upon request.
FAQs