Following evaluation of a dossier submitted by Pharming Group N.V. last year, the Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion to the European Commission on the company’s request to include the treatment of HAE attacks in adolescents with HAE and to remove the requirements for rabbit IgE testing that forms part of the EU label for Ruconest.
Following adoption of the CHMP opinion by the European Commission, this will mean that adolescents with HAE now also have access to (non-blood derived) recombinant C1- inhibitor therapy for the treatment of their angioedema attacks. In addition, the requirement to test HAE patients for pre-existing antibodies against rabbit dander, prior to treatment with Ruconest and following each tenth treatment with Ruconest, will be removed from the label. The requirement for IgE testing was a specific EU request based on a single adverse drug reaction in a study subject, who did not disclose the pre-existing rabbit allergy prior to rhC1Inh treatment. The need for testing was not required in the US as more safety data were available at the time of the Biologics License Application (BLA) and subsequent FDA-approved label in 2014.
Prof. Bruno Giannetti, MD, PhD, Pharming’s COO commented: “This EU label change will now also give adolescent in the EU the long awaited access to treat their HAE attacks with a non-blood derived C1-inhibitor and in addition, the positive CHMP opinion confirms the well-established safety profile of Ruconest, based on a database of a dozen controlled clinical trials as well as more than 12,000 post-marketing doses of Ruconest provided to HAE patients. After adoption of the CHMP opinion by the European Commission, this will remove the burden on patients and doctors to perform testing prior to and after treatments with Ruconest and enable emergency treatment with Ruconest® for HAE attacks in previously untreated HAE patients. HAE patients previously dependent on plasma derived C1INH therapies, including adolescents, will be able to receive Ruconest with the benefit of eliminating risks of exposure to known blood borne pathogens, such as Hepatitis A, B, C, E, HIV, and CJD, as well as continuously (re)-emerging pathogens, such as the recent ZIKA virus.”