EMA Approves New Production Facility for Ruconest, Pharming’s Treatment for Acute HAE Attacks

EMA Approves New Production Facility for Ruconest, Pharming’s Treatment for Acute HAE Attacks

The European Medicines Agency (EMA) has approved a type II variation for a new facility to produce Ruconest (conestat alfa), Pharming’s lead therapy for acute attacks in hereditary angioedema (HAE).

The new facility is expected to be fully operational by 2021 and to increase the production capacity of Ruconest to meet its high demand in Europe.

“As we continue to see increasing demand for Ruconest in the treatment of hereditary angioedema, we are pleased to announce the approval of our new facility, which will enable us to significantly increase production capacity for supply to patients in the EU,” Sijmen de Vries, chief executive of Pharming, said in a press release.

“In addition, as a result of our recent re-acquisition of Ruconest’s European distribution rights from Sobi, this capacity expansion will allow us to reach an even greater number of EU patients,” he added.

In the U.S., the distribution of Ruconest produced in the new facility is still being reviewed by the U.S. Food and Drug Administration (FDA). Approval is expected in the coming months.

Ruconest is a recombinant human C1-inhibitor (rhC1-INH) that works by replacing the missing C1-INH protein in HAE patients, thereby easing bouts of acute swelling triggered by the lack of C1-INH in their blood.

The therapy is currently approved in dozens of countries worldwide to treat acute swelling episodes in adults with HAE, as well as adolescents in the U.S., Israel, South Korea, and Europe.

A recent open-label Phase 2 trial (NCT01359969) evaluated the safety and efficacy of Ruconest at controlling acute HAE attacks in young children up to the age of 14.

The study enrolled 20 children, ages 5–14, who received intravenous Ruconest at a dose of 50 U/kg to treat a total of 73 HAE acute attacks. More than a third (35.0%) of the children received Ruconest for four or more acute attacks.

Results showed that a single dose of Ruconest was sufficient to stop 95.9% of the attacks.

Within one hour after receiving Ruconest, children started feeling relief; and within two hours (122.5 minutes), swelling was minimal in all body parts affected by the attack. These values were consistent across all attacks treated with Ruconest in the trial.

Treatment was found to be generally safe and well-tolerated. No serious adverse events were reported, and none of the children discontinued treatment due to side effects.

Additional trials suggest that Ruconest could also work as a preventive therapy for people with HAE. Pharming had asked the FDA to expand Ruconest’s label to include routine prevention of swelling attacks caused by HAE, but the agency rejected the request, claiming that an additional clinical trial is needed to confirm the treatment’s benefits.