Pharming prepares to appeal ruling on European marketing authorization application for Rhucin
LEIDEN, THE NETHERLANDS -- 12/13/07 -- Biotech company Pharming Group NV ("Pharming" or "the Company") (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) has adopted a negative opinion on the Marketing Authorization Application (MAA) for RhucinÂ®. On the basis of currently available information, Pharming understands that the CHMP was not reassured that there was sufficient evidence to confirm the benefits of Rhucin in laryngeal sub-mucosal attacks, or how safe and effective the medicine is when given to a patient more than once. Pharming intends to apply, later this month, for a re-examination of the CHMP opinion in accordance with relevant procedural guidelines of the EMEA. A final opinion of the CHMP after re-examination is expected in the first half of 2008.
Earlier in 2007, Pharming announced the positive results of an interim analysis of a European placebo-controlled trial in which RhucinÂ® was shown to be safe and effective in treating acute attacks of Hereditary Angioedema (HAE). The Independent Data Monitoring Committee (IDMC) reviewed these results and recommended that the Company discontinue further randomized treatments for both ethical and methodological reasons.
Dr. Bruno Giannetti, Chief Operations Officer of Pharming, commented: "We disagree with the opinion of the CHMP as the findings of the clinical studies have demonstrated clear evidence of RhucinÂ®'s efficacy in treatment of acute HAE attacks. We have been able to show that RhucinÂ® acts quickly and that none of the patients treated so far have experienced a relapse of HAE-attack or any treatment related adverse events. Until today, the product has been administered more than 150 times to human subjects of which several received 5 or more doses of RhucinÂ®. Due to the fact that HAE is an orphan disease, the number of patients treated during clinical programs is relatively low, but we remain convinced that the data submitted to the CHMP provide a basis for the granting of a marketing authorization. The quality of the product and the clear efficacy and safety data shown in the European randomized clinical trial suggest that RhucinÂ® is an advance in the many markets of Europe where no product has been approved to date.
We intend to vigorously pursue our case for approval and to continue to work with the authorities to make this product available to the patients in the shortest timeframe possible. In many European markets there is currently no registered product available to effectively treat this serious disease. We are committed to satisfy this high medical need.
While today's ruling by the CHMP will cause a delay in getting RhucinÂ® to HAE-patients in Europe, it does not change the Company's plans nor its strategy. We will continue to build on our product portfolio and our technology platforms backed by a sound financial position."
Recently, another placebo-controlled trial was completed in North America and top-line results are expected to become available in the near future. 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. In total, 55 acute attacks of HAE were treated. While the analysis of the placebo-controlled trial will be finalized soon, the results of the open-label treatments were positive and in line with earlier published data.
Conference Call Information
Pharming will discuss this opinion and the status of RhucinÂ® in a conference call for media and analysts at 17:30 CET today. An audiocast of the conference call will be available on Pharming's website shortly 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 "Rhucin-update". Mr. Rein Strijker will be chairman of the conference call.
About RhucinÂ® and HAE
RhucinÂ® (recombinant human C1 esterase inhibitor) is a human protein developed through Pharming'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® (recombinant human C1 inhibitor) for Hereditary Angioedema 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 technologies in the field of tissue repair (via its collaboration with Novathera) and 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.