Ruconest for Acute HAE Attacks Now Reimbursable in Spain

Spain has added Ruconest (conestat alfa), Pharming’s treatment for acute attacks of hereditary angioedema (HAE), to its list of reimbursable medications.

“We are delighted with this positive reimbursement decision by the Spanish Ministry of Health, as it means patients in Spain in need of new treatment options for hereditary angioedema will now be able to access Ruconest,” Sijmen de Vries, CEO of Pharming, said in a press release.

“We look forward to working with the Spanish healthcare community to ensure a rapid rollout of the product,” de Vries added.

Ruconest, a lab-made version of human C1-INH, is intended to act as a replacement for the protein in people with HAE, thereby reducing swelling. Acute attacks can become life-threatening when it affects the airways.

“AEDAF, the Spanish HAE Patient Association, welcomes the approval of new treatments which might help alleviate the suffering of patients with HAE,” said Sarah Smith, president of the association.

HAE is caused by the lack of a protein called C1 esterase inhibitor (C1-INH). Its absence triggers a cascade of events that ultimately lead to the widening of blood vessels, fluid leakage, and, as a consequence, swelling.

While preventive (prophylactic) therapies can help reduce the frequency of HAE attacks, these episodes may still occur, hence the need for “on-demand” treatments.

The protein in Ruconest’s composition is isolated from rabbit’s milk, rather than human blood. For that reason, it carries no risk of infection to patients and is more easily accessible.

The therapy is currently approved in the U.S., Europe, and other regions as an on-demand treatment for acute HAE attacks in adults and adolescents. The European Commission also approved its use in children, 2 and older.

The commission’s decision to approve Ruconest was based on data from an open-label Phase 2 clinical trial (NCT01359969), which evaluated the safety and effectiveness of the therapy in 20 children, ages 2–13.

Results showed that a single dose of Ruconest was sufficient to stop nearly all HAE attacks (95.9%). The three attacks that did occur were controlled after a second dose was administered. The median time to symptom relief after treatment was 60 minutes.

Additional data from a placebo-controlled Phase 2 trial (NCT02247739) involving 32 adolescents and adults, and a more recent pilot study of 25 adults (NCT00851409) suggested that Ruconest is an effective short-term preventive therapy for angioedema attacks.

In the U.S. and Europe, patients can have a self-administration kit at their disposal, which allows them to inject Ruconest when they feel a swelling attack starting. Appropriate training is required.