The European Commission has approved an extension in the indication of Ruconest’s (conestat alfa) Marketing Authorisation to include the treatment of acute angioedema attacks in children with HAE. This marketing authorisation expands the age range of Pharming Group N.V.’s lead product, Ruconest, a recombinant analogue of human C1 esterase inhibitor. Ruconest was previously approved for adults and adolescents in Europe.
The European Commission’s decision allows children aged two years and older to be treated with Ruconest for acute angioedema attacks. In the European Union, Ruconest has been approved for this indication in adults since 2010 and in adolescents since 2016.
The C1 esterase inhibitor protein is required to control the ‘complement’ and ‘contact’ systems, collections of proteins in the blood that fight against infection and cause inflammation. Patients with low levels of this protein have excessive activity of these two systems, which leads to the symptoms of angioedema. The active substance in Ruconest, conestat alfa, is a copy of the C1 esterase inhibitor protein and works in the same way as the natural human protein. When it is given during an angioedema attack, Ruconest stops this excessive activity, helping to relieve the patient’s symptoms.
Sijmen de Vries, CEO of Pharming, says:
“We are pleased to receive approval from the European Commission and to be able to offer Ruconest as a treatment for acute HAE attacks in all patients aged two years and above. As we expand our distribution network in Europe following the reacquisition of Ruconest-licensed territories in December 2019, we are seeing increasing demand for the product in the treatment of HAE. This approval allows us to treat the most vulnerable patients and further demonstrates the safety and efficacy of Ruconest.”