Ruconest (rhC1INH) is a recombinant human C1 inhibitor (C1INH) by Pharming, approved to treat acute attacks of hereditary angioedema, a rare condition that results from dysfunctional or low levels of C1INH in the blood.
A self-administration kit is also approved in both the U.S. and the European Union, allowing patients to inject themselves with Ruconest when they feel a swelling attack starting. Appropriate training is required.
How does Ruconest work?
C1INH controls the C1 protein, one of the molecules in the complement system. The complement system is a part of the immune system and functions as a support mechanism for immune responses against infections. More than 30 proteins, which circulate in the bloodstream in their inactive form, make up the complement system.
When there is an infection, these proteins sequentially get activated in a cascade: the first protein of the cascade activates the second, the second activates the third, and so on. There are three ways to activate the complement system: classical, lectin-mediated, and alternative. The first molecule of the classical way is C1, which an antibody-antigen complex that activates due to an infection.
In hereditary angioedema, poorly working or insufficient levels of C1INH in the blood cause fluid leakage and swelling. Conestat alfa, the active compound in Ruconest, is a recombinant human C1INH that is produced in rabbits and isolated from their milk. As opposed to C1INH that is isolated from human donors, Ruconest does not constitute an infection risk to a patient, or pose accessibility problems based on the availability of donor blood.
Ruconest in clinical trials
The European Medicines Agency (EMA) approved Ruconest in 2010 to treat adults — and, later, teenagers as well — based on positive results from three clinical studies (NCT00225147, NCT01188564, and NCT00262301). U.S. Food and Drug Administration (FDA) approval followed in 2014, covering adults and teenagers with acute symptoms of hereditary angioedema.
Researchers also tested Ruconest for its efficiency in preventing acute hereditary angioedema attacks. An international Phase 2 study (NCT02247739) found that regular injections of Ruconest resulted in significantly less frequent attacks.
Results of a Phase 2 clinical trial (NCT01359969), which concluded in 2017, also showed that Ruconest is safe in children, ages 2 to 13. The European Commission extended Ruconest’s EU approval to include children starting at age 2 in March 2020.
Ruconest is available for use, by trained patients or caregivers, as a self-administration kit in the U.S. and the EU.
The most common side effects of Ruconest are headaches, diarrhea, and nausea. Uncommon side effects may include tingling, prickling, or numbness in the skin and limbs, dizziness, throat irritation, abdominal pain, and hives.
Last updated: May 8, 2020
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