Leiden, The Netherlands, and Stockholm, Sweden October 28, 2010. Biotech company Pharming Group NV (Pharming or “the Company”) (NYSE Euronext: PHARM) and Swedish Orphan Biovitrum (Sobi) (STO: SOBI) today announced that the European Commission has granted Pharming Marketing Authorization for its lead product Ruconest™ for the treatment of acute attacks of Hereditary Angioedema (HAE). Pharming will now receive a €5 million milestone payment from marketing and distribution partner Sobi.
Following the unanimous positive opinion adopted by the Committee for Medicinal Products for Human Use (CHMP), Ruconest’s Marketing Authorization has now been ratified by the European Committee.
Patients suffering from HAE experience unpredictable, painful and debilitating attacks, due to reduced levels of C1 inhibitor, resulting in intense swelling of parts of the body (for example, face, throat, abdomen) which can last up to five days if left untreated. Ruconest is a recombinant version of the human C1 inhibitor protein, produced by Pharming’s proprietary transgenic technology. As published in last October’s issue of the Journal of Allergy and Clinical Immunology, Ruconest has been shown to have excellent efficacy and safety. Pharming has withdrawn the orphan status application in order to avoid delays in commercializing the product.
Dr Marco Cicardi, MD, Professor of Internal Medicine at the University of Milan, Italy, said: “Patients with HAE experience an average of eight attacks a year. This distressing and potentially life-threatening condition requires new approaches for acute treatment and Ruconest offers this. This recombinant product is the only HAE treatment achieving circa 90% of success in treating attacks.”
Ruconest is now approved for use in the 27 EU countries plus Norway, Iceland and Liechtenstein. Sobi will launch Ruconest initially in Germany, the UK, Sweden, Finland and Denmark, followed by a rolling launch in each of the other European Area countries.
Sijmen de Vries, CEO of Pharming: "The granting of marketing authorization for Ruconest™ is a very important step in Pharming’s evolution from a drug development company to a commercial specialty pharmaceutical business. Together with our marketing partners, we look forward to the launch of Ruconest this year and making this novel new treatment available to the European HAE population. I would also like to thank the many European physicians and patients who participated in our clinical trial program with Ruconest.”
“Ruconest has the potential to offer patients with significant medical needs a safe and valuable alternative treatment. Moreover, it is a good fit with Sobi’s commercial portfolio of rare disease products. We are very much looking forward to exciting fruitful partnership with Pharming and the opportunity to provide this new exciting product to patients who need it in Europe,” said Martin Nicklasson, CEO of Sobi.
Conference call information
Pharming’s management team will be available for questions on today’s marketing approval of Ruconest in a conference call tomorrow, October 29, at 8:30 am 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
An audio cast of the conference calls will be available on Pharming's website shortly thereafter.
About Ruconest (Rhucin in non-European countries)
Ruconest (INN conestat alfa) is a recombinant version of the human C1 inhibitor (C1INH) protein. The product is produced through Pharming’s proprietary technology in milk of transgenic rabbits. 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.
The European public assessment report (EPAR) for Ruconest will be published on the EMA website. It explains how the CHMP assessed Ruconest and its recommendations on the conditions for use of Ruconest.
About Hereditary Angioedema
HAE is a human genetic disorder in which the patient is deficient in or lacks a functional plasma protein C1 inhibitor, resulting in an overreaction of the immune system. The disease is characterized by unpredictable and debilitating episodes of intense swelling of the extremities, face, trunk, genitals, abdomen and upper airway, which may last up to five days when untreated. In addition to the life-threatening nature of the disease in case of laryngeal attacks, quality of life for individuals with the disease may be seriously impaired. Approximately one in 30,000 individuals (1:10,000 – 1:50,000) suffers from HAE with an average of approximately eight acute attacks per year.
About Swedish Orphan Biovitrum
Swedish Orphan Biovitrum is a Swedish based niche specialty pharmaceutical company with an international market presence. The company is focused on providing and developing specialist pharmaceuticals for rare disease patients with high medical needs. The portfolio consists of about 60 marketed products and an emerging late stage clinical development pipe-line. Our focus areas are: hemophilia, inflammation/autoimmune diseases, fat malabsorption, cancer and inherited metabolic disorders. Swedish Orphan Biovitrum had pro-forma revenues 2009e of about 2 BSEK and approximately 500 employees. The head office is located in Sweden and the share (STO: SOBI) is listed on NASDAQ OMX Stockholm. For more information please visit www.sobi.com.
About Pharming Group NV
Pharming Group NV is developing innovative products for the treatment of genetic disorders, specialty products for surgical indications, and nutritional products. Pharming’s lead product Ruconest™ (Rhucin in non-EU territories) has Market Authorization in the European Economic Area. The product is also under development for follow-on indications, i.e. antibody-mediated rejection (AMR) and delayed graft function (DGF) following kidney transplantation. The technologies of the Company include innovative platforms for the production of protein therapeutics, including technology and processes for the purification and formulation of these products. Additional information is available on the Pharming website 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.
Swedish Orphan Biovitrum may be required to disclose the information provided herein pursuant to the Swedish Securities Markets Act. The information was provided for public release on October 28, 2010 at 16:30 a.m. CET.
For more information please contact:
Pharming Group NV
Marjolein van Helmond, T: +31 (0)71 52 47 431 or +31 (0)6 109 299 54 or E: email@example.com
Swedish Orphan Biovitrum
Erik Kinnman, VP Investor Relations, T: +46 73 422 15 40 or E: firstname.lastname@example.org
Martin Nicklasson, CEO, T: +46 8 697 20 00
Julia Phillips, T: +44 20 7269 7187