Pharming plant gets EMA approval, easing Ruconest supply pressures

Rising demand for Ruconest (onestat alfa), a treatment for hereditary angioedema (HAE) attacks, created the need for more capacity at Pharming, a specialty pharmaceutical company. Pharming responded by building a second production plant in the Netherlands but had to wait for European Medicines Agency (EMA) approval before shipping supplies from the new site.

The EMA has now signed off on the Dutch plant, clearing Pharming to ship products it manufactured during the approval process and thereby providing a boost to efforts to meet demand for Ruconest in the European Union (EU).

Talking to investors last year, Sijmen De Vries, the chief executive of Pharming, framed the opening of the site as fully resolving the question of how to service the HAE market.

De Vries said, “The first site will be very sufficient to actually satisfy the needs for Ruconest production [in] HAE​.”

The new capacity has become available at a time when Pharming thinks it is on the cusp of another rise in the sales of Ruconest in the EU. Earlier this month, Pharming took back commercial rights to the drug in 36 countries, including all remaining EU markets, from Swedish Orphan Biovitrum.

Based on what happened when it regained the North American rights from Valeant Pharmaceuticals late in 2016, Pharming expects its decision to take control of EU marketing to drive sales growth.

In parallel to the EU sales push, Pharming will work to get the new plant approved by the US Food and Drug Administration (FDA). Pharming expects the US FDA to approve the facility in the first half of this year, at which point the speciality pharmaceutical company will be cleared to use the site to ship products to its two key markets.

Pharming is using its rabbit platform to serve the EU and US HAE markets. The platform is based on transgenic rabbits that produce milk containing the recombinant protein used in Ruconest.

With the new facility now EMA approved, Pharming thinks it has enough rabbit-based capacity to meet the demands of patients with HAE, a rare disease. However, Pharming will need to change its approach if it is to expand into diseases that affect more people.

De Vries said, “For the future, of course, the long-term future, we have reinstalled the cattle platform because the numbers of vials necessary for any of the new indications, [acute kidney injury] and pre-eclampsia, the rabbit platform will be unpractical​.”