Elapegademase

Elapegademase (INN) is a recombinant enzyme used for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID). It is a form of enzyme replacement therapy that is marketed under the brand name Revcovi.

Mechanism of Action

Elapegademase works by replacing the missing or non-functioning adenosine deaminase enzyme in patients with ADA-SCID. This enzyme is crucial for the breakdown of adenosine and deoxyadenosine, which are toxic to lymphocytes if they accumulate. By breaking down these substances, elapegademase helps to restore the immune system's function.

Medical Uses

Elapegademase is used for the treatment of ADA-SCID in both pediatric and adult patients. It is administered via intramuscular injection.

Side Effects

Common side effects of elapegademase include cough, vomiting, rash, and fever. Serious side effects may include allergic reactions and infection due to the immune system's compromised state.

History

Elapegademase was approved for medical use in the United States in 2018. It was developed by Leadiant Biosciences, and is the second enzyme replacement therapy approved for ADA-SCID, following pegademase.

See Also

• Enzyme replacement therapy
• Adenosine deaminase deficiency
• Severe combined immunodeficiency

References

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