Pharming Group Drug Gets Special FDA Status, Shares Jump
Leiden, The Netherlands, October 11, 2007. Biotech company Pharming Group N.V. announced today that it has received Orphan Drug designation for recombinant human fibrinogen (rhFIB) from the US Food and Drug Administration (FDA) for the treatment of bleeding in patients deficient in fibrinogen. Pharming has initiated development of recombinant human fibrinogen as a pharmaceutical product for genetic and acquired deficiencies with the orphan designation and will continue to pursue medical device development using rhFIB through partnerships.
Recombinant human fibrinogen is being developed as a replacement therapy to treat bleeding in patients with fibrinogen deficiency. Fibrinogen is a critical protein involved in the clotting of blood. Consequently, deficiency of fibrinogen can result in uncontrolled bleeding. Bleeding resulting from low levels of fibrinogen develops in numerous clinical settings including trauma, surgery, liver disease, sepsis, and cancer.
In the United States, approximately 100,000 patients develop bleeding episodes each year due to a fibrinogen deficiency. Replacement of fibrinogen in these patients can result in normal hemostasis. Current standard of care for patients in the United States who are deficient in fibrinogen is the use of impure human blood products such as cryoprecipitate, which is composed mainly of fibrinogen. In select countries in Europe, plasma-derived fibrinogen is used to treat such bleeding.
Dr. Francis Pinto, Chief Executive Officer of Pharming commented, "Pharming's development strategy for rhFIB will utilize the successful model of recombinant blood clotting proteins to control bleeding for various genetic and acquired deficiencies. As Rhucin® nears its first market authorization, rhFIB represents another avenue of growth for Pharming and demonstrates our strength in developing therapeutic recombinant proteins for unmet medical needs."
In addition to the protection provided by the Orphan Drug designation, Pharming has a portfolio of patents covering transgenic production, purification and use of various proteins, including recombinant human fibrinogen. The resulting intellectual property position provides Pharming broad protection on rhFIB for pharmaceutical and medical device applications.
Background on Orphan Drug Designation and Recombinant Human Fibrinogen
The FDA Orphan Drug designation is reserved for promising new therapies being developed to treat diseases that affect fewer than 200,000 people in the United States. The designation provides an accelerated review process, tax advantages and a seven-year period of market exclusivity in the US upon product approval.
Pharming is developing recombinant human fibrinogen as a pharmaceutical product for genetic and acquired deficiencies of fibrinogen. The existing market size for fibrinogen deficiencies is estimated to be over USD 500 million in the developed world. Current standard of care in the United States for patients deficient in fibrinogen is the use of cryoprecipitate, which is composed mainly of fibrinogen. In select countries in Europe, a plasma fibrinogen product is marketed. With pharmaceutical development ongoing for rhFib, Pharming will continue to pursue partnerships with medical device manufacturers to build further value for its fibrinogen franchise.
Over the last few years, Pharming has been building a program on biomaterials through collaborations with the BioMedical Materials consortium (BMM), Novathera Ltd and other institutions. Pharming has successfully produced high levels of recombinant human fibrinogen using its production technology. Human fibrinogen is a natural blood protein that can form insoluble fibrin poymers to stop bleeding. In laboratory tests and initial animal studies, rhFIB has been demonstrated to be virtually identical in structure and function to plasma fibrinogen. The Company has already provided rhFIB for evaluation to device manufacturers and research institutions to facilitate novel product development for various applications.
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 (MAA under review by EMEA) and human lactoferrin for use in food products (GRAS notification under review by 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). 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. The press release also appears in Dutch. In the event of any inconsistency, the English version will prevail over the Dutch version.