The Committee for Medicinal Products for Human Use (CHMP) of the European Medicine Agency (EMA) has recommended approval of Ruconest for the treatment of acute angioedema attacks in children with HAE. This marketing authorization would expand the age range of Pharming Group N.V.‘s lead product, a recombinant analogue of the human C1 esterase inhibitor produced by recombinant DNA technology in the milk of transgenic rabbits.
The positive opinion is based on the data from a Phase II clinical trial in 20 children. This recommendation will allow children aged 2 years and older to be treated with Ruconest for acute angioedema attacks. The European Commission’s (EC) approval decision is expected in June 2020. In the European Union, Ruconest has been approved for this indication in adults since 2010 and in adolescents since 2016.
In January 2020 Pharming received EMA approval for a new facility. Together with this extension of the indication, Pharming believes that supply of Ruconest to the market is now further strengthened. Ruconest will be available for use in children patients later in 2020 throughout Member States of the EU, as well the European Economic Area in which Pharming currently has a license in the adult and adolescent population. Sijmen de Vries, CEO of Pharming, says:
“As we continue to see increasing demand for Ruconest in the treatment of HAE, we are pleased to announce this step forward in this paediatric label expansion. This will enable us to treat attacks of patients of all ages living with HAE in the EU. In addition, as a result of our recent re-acquisition of Ruconest’s European distribution rights from Sobi, this extension of the indication will allow us to reach an even greater number of EU patients.”
The open-label, single arm, Phase II clinical trial was designed in agreement with the EMA as part of a Paediatric Investigation Plan (PIP) to assess the pharmacokinetic, safety and efficacy profiles of Ruconest at a dose of 50 U/kg in HAE patients aged 2-13 years in support of the indication for treatment of HAE attacks in children.
A total of 20 children with HAE were treated for 73 HAE attacks at a dose of 50 U/kg (up to a maximum of 4200 U). The study reported clinically meaningful relief of symptoms assessed using a visual analogue scale (VAS) completed by the patient (assisted by their parent). The median time to onset of relief was 60 minutes (95% confidence interval: 60-653), and the median time to minimal symptoms was 123 minutes (95% confidence interval: 120-126). Only 3/73 (4%) attacks were treated with a second dose of Ruconest.
Ruconest was generally safe and well-tolerated in the study. No patients withdrew from the study due to adverse events. There were no related serious adverse events, hypersensitivity reactions, or neutralizing antibodies detected.