CHMP recommends extending indication of Ruconest to children with Hereditary Angioedema.- Pharming

Pharming has announced the Committee for Medicinal Products for Human Use (CHMP), an advisory committee of the European Medicine Agency (EMA), has adopted a positive opinion recommending an extension of the indication for Ruconest (recombinant human C1 esterase inhibitor) to the European Commission for treatment of acute angioedema attacks in children with Hereditary Angioedema.

This marketing authorisation would expand the age range of Pharming’s lead product, Ruconest, a recombinant analogue of the human C1 esterase inhibitor produced by recombinant DNA technology in the milk of transgenic rabbits. Ruconest was previously approved for adults and adolescents in Europe.

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 (EU), Ruconest has been approved for this indication in adults since 2010 and in adolescents since 2016.