Pharming Group to Request New Approval for Rhucin Drug
In accordance with European regulation (EC) No 726/2004, Pharming requested a re-examination of the grounds for refusal that led to a negative opinion by the Committee for Medicinal Products for Human Use (CHMP), regarding the granting of Market Authorization of Rhucin® for treatment of attacks of Hereditary Angioedema (HAE) in the European Union. Based on these grounds for refusal, the Company will submit detailed grounds for this re-examination request to the EMEA no later than February 11, 2008 (within sixty calendar days after receipt of the negative opinion).
In the re-examination procedure, the CHMP has appointed a new Rapporteur and co-Rapporteur, who will assess the dossier, in particular the elements that form the basis of Pharming's grounds for the request. The Rapporteur and co-Rapporteur will report their findings to the CHMP, to which the Company can respond. The CHMP will issue their final opinion at a CHMP meeting within sixty calendar days following receipt of the grounds for the request. This opinion will form the basis of a decision on the Market Authorization of Rhucin® within the EU. Therefore, such a final decision can be expected in the second quarter of 2008.
Pharming believes that the results obtained in clinical studies form the basis for a positive risk benefit ratio for Rhucin® in treatment of acute HAE-attacks. In a randomized placebo-controlled study, Rhucin® showed statistically significant superiority over placebo in time to first relief of the attack and in time to minimal symptoms. Efficacy was shown earlier in open-label clinical trials. No patient experienced a relapse of the HAE attack or any treatment-related adverse event. Once authorized to enter the market, Rhucin® would make an additional replacement therapy available throughout the EU.
In the US, a double-blinded randomized placebo-controlled clinical trial was completed in the fourth quarter of 2007. When the last patients complete their follow-up visits, the data will be analyzed by an independent statistician. The Company expects to have summary data available later this quarter. The US open-label study is still ongoing. Depending on the outcome of the placebo-controlled study and a pre-BLA (Biologic License Application) meeting with the FDA, the Company anticipates to file the application for a US market approval in 2008.
While the regulatory process of Rhucin® is still ongoing, Pharming continues its preparations for the commercialization of the product. The clinical database is being expanded by continued inclusion of patients in open label studies, of which the resulting data should eventually support marketing efforts. In addition, the commercial protection of Rhucin® has been further strengthened by the recent issuance of European Patent 1252184 entitled "C1 inhibitor produced in the milk of transgenic mammals". The claims of this patent cover production, purification and the use of C1 inhibitor (the scientific name of Rhucin®) in pharmaceutical preparations.
About Rhucin® and HAE
Rhucin® (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. Rhucin® 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 - Rhucin® (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: www.pharming.com and on 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.
Contact:
Carina Hamaker (NL) Julia Philips (UK) Investor Voice Financial Dynamics T: +31 (0)6 537 499 59 T: +44 (0)20 7269 7187 T: +31 (0)71 52 47 400 T: +44 (0)7770 827 263
Rein Strijker Samir Singh (US) Pharming Group NV Pharming Healthcare Inc T: +31 (0)71 52 47 400 T: +1 908 720 6224
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