Pharming Group Receives Positive Opinion From European Medicines Evaluation Agency On Rhucin
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.”
Conference call information
Chief Executive Officer Sijmen de Vries will discuss this opinion on Ruconest and next steps in a conference call for press and analysts at 5.15 pm CET. To participate in the call, please call one of the following numbers: - From the Netherlands: 0800 265 8543 (toll-free) or +31 (0)45 631 6901 - From the UK: 0800 358 0886 (toll-free) or +44 207 153 2027
Following a short introduction, the lines will be opened for a question and answer session. An audio cast of the conference calls will be available on Pharming's website shortly thereafter.
About Ruconest (Rhucin)
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.
About Hereditary Angioedema
Hereditary Angioedema (HAE) is a debilitating and potentially life-threatening genetic condition, resulting in spontaneous and recurring attacks of angioedema. Angioedema attacks are characterized by swelling of soft tissue in a reaction visually similar to severe allergic reactions. Attacks may affect various locations such as the intestines, the face, extremities or mouth and throat. Without treatment, attacks progress during the first 24 hours and then subside over another two to five days. Attacks are painful and disfiguring and cause significant morbidity. In addition to the life-threatening nature of the disease, quality of life for individuals with the disease may be seriously impaired.
HAE is caused by the lack or deficiency of endogenous C1INH activity, which results in an overreaction of the immune system. Patients typically present in late childhood, with the mean onset at 11 years. The prevalence of HAE is approximately 1 in 30.000 individuals. The frequency of attacks varies significantly in the affected population. Current treatment options include prophylactic treatment with tranexamic acid and androgens, which may reduce the frequency of attacks. The average frequency even under such anabolic steroid prophylaxis is still estimated to be 6-8 treatment requiring attacks per year. For the treatment of acute attacks, the current standard of care in Europe is plasma derived C1 inhibitor from human donors. Swedish Orphan Biovitrum International estimates the current value of the European HAE market to approximately €110 million and sees the potential of the HAE market to increase with the introduction of Rhucin® as one of several new treatment alternatives.
About Pharming Group NV
Pharming Group NV is developing innovative products for the treatment of genetic disorders, ageing diseases, specialty products for surgical indications, and nutritional products. Following a positive opinion from the European authorities (EMA’s CHMP), Pharming’s lead product Ruconest (Rhucin) for acute HAE attacks is awaiting the EU Marketing Authorization. The product is also under development for follow-on indications, i.e. antibody-mediated rejection (AMR) and delayed graft function (DGF) following kidney transplantation. Prodarsan® - a product under development by Pharming's subsidiary DNage - is in early stage clinical development for Cockayne Syndrome and lactoferrin for use in food products. The advanced technologies of the Company include innovative platforms for the production of protein therapeutics, technology and processes for the purification and formulation of these products, as well as technology in the field of DNA repair (via DNage). Recently the partial spin off of DNage was initiated. Additional information is available on the Pharming website, http://www.pharming.com.
This press release contains forward looking statements that involve known and unknown risks, uncertainties and other factors, which may cause the actual results, performance or achievements of the Company to be materially different from the results, performance or achievements expressed or implied by these forward looking statements.
Contact: Ms. Marjolein van Helmond, T: +31 (0)71 52 47 431 or +31 (0)6 109 299 54 or E: m.vanhelmond@pharming.com
Juliet Edwards Assistant Financial Communications Holborn Gate, 26 Southampton Buildings London, WC2A 1PB T +44 (0)20 7269 7125 F +44 (0)20 7242 8695
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