CHMP Favors EU Approval of Ruconest for Acute HAE Attacks in Children

CHMP Favors EU Approval of Ruconest for Acute HAE Attacks in Children
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The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, has recommended Ruconest (conestat alfa) for approval as treatment for acute swelling episodes in children with hereditary angioedema (HAE).

In the European Union, Pharming‘s Ruconest is approved for adults and adolescents with the condition. If the European Commission, which makes the final decision based on the CHMP’s recommendation, approves the medicine, it will become available to all HAE patients, regardless of age. A decision is expected in June.

“As we continue to see increasing demand for Ruconest in the treatment of hereditary angioedema, 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,” Sijmen de Vries, CEO of Pharming, said in a press release.

“In addition, as a result of our recent reacquisition 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,” de Vries said.

Mutations in the gene that provides instructions for making the C1 esterase inhibitor protein (C1-INH) are responsible for most cases of HAE. In type 1, mutations cause a deficiency in this protein, whereas in type 2, a less-effective form of C1-INH is produced.

Lack of the C1-INH protein triggers a chain of reactions that cause blood vessels to dilate and fluid to accumulate in soft tissues, triggering acute bouts of swelling in patients.

Ruconest, a lab-made version of the C1-IHN protein, is designed to replace the defective protein in HAE patients, helping treat acute swelling attacks. It is approved for adults and adolescents in the U.S., Europe, and Israel, and for adult patients in many other countries.

The decision to recommend Ruconest for children was based on findings from an open-label Phase 2 clinical trial (NCT01359969), which assessed the safety and effectiveness of Ruconest in young children, age 2–13.

Twenty participants were treated with Ruconest at a dose of 50 U/kg (up to a maximum of 4200 U) each time they experienced an acute swelling attack. In total, 73 episodes received treatment, with one-third (35%) of participants being treated for four or more attacks.

Results showed that a single dose of Ruconest was sufficient to stop 95.9% of the attacks, providing symptom relief after a median of one hour. Within a median of two hours, symptoms were minimal.

Ruconest was generally safe and well-tolerated. The most common adverse side effects included cold, vomiting, abnormal morphology of white blood cells, and viral infection. No patients experienced a serious treatment-related side effect, or withdrew from the study because of that.

The EMA and the U.S. Food and Drug Administration recently approved a new facility in the Netherlands to produce Ruconest, increasing the production capacity to meet the therapy’s high demand. The facility is expected to be fully operational in 2021.

Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Ciências e Tecnologias and Instituto Gulbenkian de Ciência. Inês currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.
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Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.
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Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Ciências e Tecnologias and Instituto Gulbenkian de Ciência. Inês currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.
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