Ruconest (rhC1INC) is a recombinant human C1 inhibitor (C1INH) used in the treatment of acute attacks of hereditary angioedema, a rare condition caused by dysfunctional or low levels of C1INH in the blood. The condition is caused by a malfunctioning immune system and results in spontaneous attacks of major swelling in the face, limbs, intestinal tract, and upper airways.
How Ruconest works
C1INH controls a protein called C1, one of the molecules in the complement system. Complement 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 activated 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 is activated by an antibody-antigen complex in case of infection.
In hereditary angioedema, dysfunctional or insufficient levels of C1INH in the blood cause fluid leakage and swelling. Ruconest is administered as an injection and rapidly relieves the symptoms of an acute swelling attack by increasing functional C1INH levels in the blood.
Conestat alfa, the active compound in Ruconest, is a recombinant human C1INH produced in rabbits and isolated from their milk. As opposed to C1INH that are isolated from human donors, Ruconest does not constitute an infection risk to the patients or have accessibility problems based on the availability of donor blood.
Ruconest in clinical trials
The European Medicines Agency (EMEA) approved Ruconest in 2010 after positive results from three clinical studies (NCT00225147, NCT01188564, and NCT00262301). This was followed by the U.S. Food and Drug Administration (FDA)’s approval in 2014 as a treatment of the acute symptoms of hereditary angioedema.
Ruconest was also tested for its efficiency in preventing acute attacks associated with hereditary angioedema. An international Phase 2 study (NCT02247739) found that regular injections of Ruconest resulted in significantly less frequent attacks.
Ruconest has been shown to be safe and effective both in adults and adolescents. Results of a recently concluded Phase 2 clinical trial (NCT01359969) showed that Ruconest is also safe for children ages 2 to 13.
Ruconest is available as a self-administration kit in the United States. The European Union has set procedures in motion to make the kit available to patients there as well.
The most common side effects of Ruconest are reported to be 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.
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