US FDA approved new drug Haegarda for the treatment of rare kind of genetic disorder termed as hereditary angioedema. FDA approved indicated that this drug should be used in adolescent and adults for the treatment of hereditary angioedema. Haegarda is basically C1 esterase inhibitor protein and it is administered via subcutaneous drug delivery system.
Hereditary angioedema is characterized as rare genetic disorder that causes swelling of extremities, face, reproductive organs and internal lining of airway and gastrointestinal tract. Hereditary angioedema may cause episodal attacks of swelling rather than appearing constantly on skin and other areas.
Exact underlying cause is still unknown but many dermatologists believe that mutations inside C1 inhibitor gene is main triggering factor is this whole phenomenon. Up-til now about more than 300 known mutations have identified is this gene. These mutations bring down the level of C1 inhibitor protein that keeps the inflammatory mediators in control like bradykinin, leukotrienes, histamine etc.
In some cases conditions may become worse due narrowing of airways. This is very serious condition and patient should go to hospital for respiratory support because breathing is almost difficult due to narrowing of airways and internal lining of lungs.
Haegarda is basically C1 esterase inhibitor protein derived from human plasma that maintains its level in case of its deficiency due to mutations in C1 gene. This drug is effective routine attacks of angioedema but not for acute attacks of angioedema.
Haegarda Side effects
According to health authorities following side effects were reported during its clinical trails
- Anaphylactic reactions (hypersensitivity)
- Swelling of nasal lining
- Feeling of swollen throat
- Itchiness at injection site
- Redness of skin
- Mild Fever in some cases
Haegarda Uses or Prescribing Information
US FDA approved Haegarda for the treatment of hereditary angioedema in adolescent and adults. This drug should only be used in routine attacks not for acute attacks. It is administered via subcutaneous route. Test dose should be to rule out any possibility of allergic reaction.
Haegarda is formulated as vial of 2000IU or 3000IU in form of lyophilized powder for reconstitution. However, its dosage frequency is 60IU/Kg subcutaneously two times in a week for the Prophylaxis of hereditary angioedema in adults.