Biotech company Pharming Group NV (Pharming) announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion on Ruconest (Rhucin) for the treatment of acute angioedema attacks in patients with Hereditary Angioedema (HAE). With this positive opinion, the CHMP recommends the European Commission to grant the European Marketing Authorization (MA). The product will be marketed in the European Union (EU) under the name Ruconest.

After review of the dossier, the CHMP has concluded that there is a favourable benefit to risk balance for Pharming’s product. It was also concluded that the name Rhucin may lead to confusion with a similarly sounding product marketed in some EU countries. Rhucin will therefore be marketed in the EU as Ruconest. The CHMP will prepare an assessment report (EPAR) including the reasons for the CHMP opinion and details of the evaluation. This report will be published on the EMA website after the granting of the MA by the European Commission, which is expected early September 2010.

Following the granting of the MA, Ruconest will be authorised for marketing in all 30 countries of the European Economic Area (the EEA includes all 27 EU countries plus Norway, Iceland and Liechtenstein). Pharming has marketing and distribution partnerships in place covering all countries of the EEA: Laboratorios del Dr Esteve for Spain, Portugal, Greece and Andorra, and Swedish Orphan Biovitrum International (SOBI) for all other European countries. SOBI is preparing the imminent launch of Ruconest in Germany and the UK. Simultaneously, pricing and reimbursement discussions in the other European Area countries will be initiated. SOBI estimates the current value of the European HAE market at approximately €110 million and sees the potential of the HAE market to increase with the introduction of Ruconest as a new treatment alternative. Upon the granting of the MA, SOBI will pay to Pharming an undisclosed milestone payment.

“The CHMP’s positive opinion for Ruconest is a landmark event for our Company. We have now made the final step towards validation and commercialization of our proprietary technology. Ruconest will be the first recombinant biopharmaceutical product from this platform to be approved in Europe and I am very proud that we have achieved this major milestone,” said Dr. Bruno Giannetti, chief operations officer of Pharming. “We are looking forward to be able to make Ruconest available to HAE patients in the EEA. Following European approval, health care professionals will have an innovative and highly effective product with a favorable safety profile to treat patients with hereditary angioedema.”

Ruconest (INN conestat alfa) is a recombinant version of the human C1 inhibitor (C1INH) protein for acute treatment of Hereditary Angioedema (HAE) attacks. The product is produced through Pharming’s proprietary technology. Ruconest has identical amino acid sequence as endogenous human C1INH. The safety and efficacy of Ruconest has been demonstrated in two placebo controlled and four open-label studies. Both randomized placebo-controlled clinical trials showed statistically significant and clinically relevant improvement in time to relief of symptoms and time to minimal symptoms compared to placebo. Ruconest holds an orphan drug designation both from the US FDA and EMA.

Pharming Group NV is developing innovative products for the treatment of genetic disorders, ageing diseases, specialty products for surgical indications, and nutritional products.