Pharming To Receive Negative Opinion On European Marketing Authorization Application For Rhucin
Company to refile marketing application and request accelerated assessmentÂ Â
Leiden, The Netherlands, March 20, 2008. Biotech company Pharming Group NV (â€œPharmingâ€ or â€œthe Companyâ€) (NYSE Euronext: PHARM) announced today that it has been informed by the European Medicines Agency (EMEA) that the Committee for Medicinal Products for Human Use (CHMP), after re-examination of its initial opinion, will adopt a negative opinion on the Marketing Authorization Application (MAA) for RhucinÂ® (recombinant human C1 inhibitor) to treat acute attacks of Hereditary Angioedema (HAE). It expects to receive a formal notofication later today. Based on this development, the Company intends to refile its marketing application for RhucinÂ® with additional data and request expedited EU regulatory review (so-called accelerated assessment).
Pharming understands that the CHMP does not have a specific concern on the safety and efficacy data submitted in the MAA and have accepted all the non-clinical and quality aspects of the product. However, it is not reassured that there is sufficient evidence to confirm the clinical benefits of RhucinÂ® in repeat use. In particular, the Committee is not reassured about the potential for undesirable immune responses following repeated administration. The re-examination procedure by EMEA included a review by an independent scientific advisory group composed of European recognized experts in the field of HAE. There was consensus among the experts, recognizing that the available clinical data were limited, that there was no evidence to indicate the development of neutralising antibodies to C1-inhibitor with repeat administration of RhucinÂ®.
The Company intends to re-submit the registration dossier with the inclusion of additional clinical data from the recently completed North American clinical study when these results become available in the second quarter of 2008. As part of the re-submisson, Pharming will request an expedited review which is an assessment period of 150 days. In the meantime the Company will pursue registration in markets outside the European Union.
Earlier in 2007, Pharming announced the positive results from an interim analysis of a European placebo-controlled trial in which RhucinÂ® was shown to be safe and effective in treating acute attacks of HAE. Recently, another placebo-controlled trial was completed in North America. In this study, 39 patients with acute attacks of HAE were administered either RhucinÂ® or placebo. In the open label follow-up phase of the trial, patients with acute attacks of HAE were eligible to receive treatments with the product. The site of attacks treated in HAE patients included laryngeal, facial, abdominal, urogenital and peripheral. While the analysis of the placebo-controlled phase remains to be finalized soon, the results of the open-label treatments were positive and in line with the previously reported findings from the European trials. Pharming intends to pursue a filing for marketing approval with the US-FDA soon after the release of results from the placebo-controlled trial.
Dr. Francis Pinto, Chief Executive Officer of Pharming, commented: â€We are disappointed by the opinion of the CHMP but will continue to work with the EMEA to address the unmet medical need of HAE patients. The findings of the clinical studies have demonstrated clear evidence of RhucinÂ®â€™s efficacy and safety in the treatment of acute HAE attacks. RhucinÂ® acts quickly and none of the patients treated so far have experienced a relapse of an HAE attack or any serious treatment-related adverse events. We remain committed to making RhucinÂ® available to European HAE patients by refiling our MAA with additional data and obtaining approval under an expedited review from EMEA. â€
Conference Call Information
Pharming will discuss this opinion and the status of RhucinÂ® in a conference call for media and analysts at 11:30 am CET today. An audiocast of the conference call will be available on Pharming's website after the call. The dial-in number from the Netherlands is 045 631 6902 . The dial-in number for outside the Netherlands is +44 207 153 2027 . Name of the conference call is â€œRhucin-updateâ€.
About RhucinÂ® and HAE
RhucinÂ® (recombinant human C1 esterase inhibitor) is a human protein developed through Pharmingâ€™s proprietary technology where the human protein is expressed in milk of transgenic rabbits. RhucinÂ® is currently under development for treatment of patients with acute attacks of Hereditary Angioedema (HAE). HAE is a human genetic disorder caused by a shortage of C1 inhibitor activity and results in an overreaction of the immune system. The disease is characterized by acute attacks of painful and in some cases fatal swelling of several soft tissues (edema), which may last up to five days when untreated.
About Pharming Group NV
Pharming Group NV is developing innovative products for the treatment of genetic disorders, ageing diseases, specialty products for surgical indications, intermediates for various applications and nutritional products. Pharming has two products in late stage development - RhucinÂ® for HAE and human 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). Additional information is available on the Pharming website, http://www.pharming.com and on http://www.dnage.nl..
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.