Pharming Receives European Orphan Drug Designation
Prevention Of Delayed Graft Function In Organ Transplantation
Leiden, The Netherlands, January 16, 2007. Biotech company Pharming Group N.V. (“Pharming” or “the Company”) (Euronext: PHARM) (PHARM.AS) announced today that it has received notice from the European Medicines Evaluation Agency (“EMEA”) that orphan drug designation (“ODD”) has been granted to recombinant human C1 inhibitor (trade name Rhucin®) for prevention of delayed graft function (“DGF”) after solid organ transplantation.
Under the guidelines of the EMEA, an ODD can be granted to a product for which it is made plausible that it will have a positive therapeutic effect on a disease which is relatively rare in the European Union. During the development phase of the new product certain incentives are given to the sponsor of the product to facilitate the registration and market authorization. These incentives include reductions of fees and an accelerated registration procedure. After the product has been proven to be safe and efficacious (by preclinical and clinical studies) and has been admitted to the market, an exclusivity of (maximally) ten years is granted to prevent the entering of the market of similar products for the same indication. Full details can be found at the website of the EMEA (http://www.emea.europa.eu/).
DGF is a serious medical condition which frequently occurs after transplantation procedures. In this condition, the transplanted organ or tissue does not function properly during the first period after the transplantation. This may be caused by an immune response of the recipient or by damage caused by oxygen or other external factors during the transplantation procedure. Current treatments include the use of immune suppressing agents. In the area of prevention much focus is given on the transplantation procedure itself and the conditions under which the organs or tissues are stored and treated during the procedure. Pharming’s product Rhucin® works in a different way than the current treatments and may, therefore, provide additional benefits.
Pharming has provided evidence to the EMEA which makes it plausible that Rhucin® will have a beneficial effect in preventing the occurrence of DGF after transplantation. This evidence was based on literature reports and on data of biological effects of Rhucin® in clinical studies and an animal model.
First clinical studies in humans, to be started after completion of the animal studies, will focus on determining the right dose in humans and aiming at demonstrating significant positive effect in preventing DGF.
Pharming believes that although the number of transplantations is currently relatively small (approximately 35,000 per year in the EU), DGF is an important medical need providing a significant market opportunity. Transplantation procedures are costly and patients often have to wait a long time before a transplantation can be performed. Reduction of the frequency of DGF will, therefore, provide a valuable medical and economical benefit.
Background on Pharming Group N.V.
Pharming Group NV is developing innovative products for the treatment of genetic disorders, specialty products for surgical indications, intermediates for various applications and food products. Pharming has two products in late stage development - recombinant human C1 inhibitor for hereditary angioedema (MAA submitted to EMEA) and human lactoferrin for use in functional foods (GRAS notification filed with FDA). 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 its acquisition of DNage BV). 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. The press release also appears in Dutch. In the event of any inconsistency, the English version will prevail over the Dutch version.
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- Samir Singh
- Pharming Group NV (US)
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