New Ruconest Manufacturing Facility Will Be Built in 2021

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by Forest Ray PhD |

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Ruconest, new manufacturing facility

Pharming Group has committed to building a new manufacturing facility in the Netherlands to expand production of Ruconest (conestat alfa), its treatment for acute attacks of hereditary angioedema (HAE).

Construction will begin in mid-2021. Once finished, it will create at least 40 new jobs, the company said.

“With an increasing demand for Ruconest for the treatment of hereditary angioedema and an increasing need for [recombinant human C1-inhibitor] in our clinical trials in new, large indications, we continue to invest in expanding our in-house processing capabilities as part of our strategy to deliver long-term growth,” Sijmen de Vries, CEO of Pharming, said in a press release.

Ruconest is a recombinant — meaning lab-made — human C1-inhibitor, or rhC1-INH, that relieves episodes of acute swelling by replacing the missing C1-INH protein that underlies attacks. This swelling can become life-threatening when it occurs in the airways.

The protein in Ruconest is produced in rabbits and isolated from their milk. As opposed to C1-INH that is isolated from human donors, Ruconest does not create an infection risk for patients, or pose accessibility problems based on the availability of donor blood.

The planned five-story, 4,000-square-meter facility won approval from the European Medicines Agency and the U.S. Food and Drug Administration earlier this year and will house infrastructure for the purification, filtration, and concentration of the raw materials for making Ruconest.

It will be located in the Pivot Park life sciences business campus in Oss, in the southern Netherlands. Pivot Park hosts more than 60 drug discovery and development companies, including BioConnection, which was contracted by Pharming to fill Ruconest vials and complete product packaging. Pharming holds a minority stake in BioConnection.

Ruconest is currently approved in dozens of countries as an on-demand treatment for acute HAE attacks in adults and adolescents. The European Commission has also approved it for use in young children, ages 2 and older.

Evidence from recent studies, a pilot study of 25 adults (NCT00851409), and a placebo-controlled Phase 2 trial of 32 adolescents (NCT02247739) and adults, all suggest that Ruconest also works well as a short-term preventive therapy for angioedema attacks.

Such preventive use included its administration prior to dental procedures, endoscopies, stressful situations, warming weather changes, and travel. It also was used following the detection of erythema marginatum, a rare skin rash of the trunk and arms and legs. In these studies, Ruconest prevented attacks in 93 of 97 instances of short-term prevention therapy among nine patients.

Pharming has previously asked the U.S. regulatory agency to expand Ruconest’s label to include routine prevention. However, the agency needs more testing to confirm the benefits seen in prior studies.

A self-administration kit is approved in both the U.S. and the European Union for patients prescribed Ruconest. It allows individuals to inject themselves with Ruconest when they feel a swelling attack starting. Appropriate training is required.