Results of phase II study for prophylaxis of HAE attacks

The results of a “Randomized, Double-Blind, Placebo-Controlled Trial of Recombinant Human C1 Inhibitor for Prophylaxis of Hereditary Angioedema Attacks” has been presented by Marco Cicardi, Professor of Internal Medicine University of Milan, Hospital L. Sacco Milan and co-prinicipal investigator for the study. The presentation was held during the American College of Allergy, Asthma and Immunology 2016 Scientific Meeting (ACAAI) meeting in San Francisco, USA.

HAE and rhC1INH:

  • HAE due to C1inhibitor deficiency is a genetic disorder characterized by episodic, unpredictable, potentially life- threatening edema and other manifestations (eg, abdominal pain)
  • rhC1INH is efficacious and well tolerated for acute HAE attacks
  • Previous open-label study suggested potential benefit of rhC1INH prophylaxis


To evaluate the efficacy and safety of rhC1INH as prophylaxis against angioedema attacks in adolescents and adults with HAE.


  • rhC1INH 50 IU/kg (up to 4200 IU/kg) administered once or twice weekly significantly reduced the number of HAE attacks
    • Most patients had ≥50% reduction during treatment with rhC1INH
    • 22 of 23 patients (95.7%) had ≥50% reduction with twice weekly rhC1INH
  • Administration of rhC1INH for up to 8 weeks was generally well tolerated
  • Further research on rhC1INH as prophylaxis for the prevention of HAE attacks is warranted

(Source: Pharming)