Netherlands based companies sign commercial manufacturing contract aimed at providing material for treatment of patients with hereditary angioedema.
Pharming Group N.V. based in Leiden has signed the agreement with DSM Biologics for purification of its lead product, recombinant human C1 inhibitor, rhC1INH, with a view to launching the product on the market.
Currently, Pharming has an agreement with Genzyme Flanders for the downstream purification of rhC1INH to provide material for an on-going Phase II clinical trial and upcoming Phase II/III clinical trial of the product.
The deal with DSM Biologics enables Pharming to transfer downstream purification of rhC1INH from Genzyme Flanders, with effect from July 1 2003, and then to start commercial production in close collaboration with DSM Biologics.
Dr. Francis Pinto, CEO of Pharming explains that the agreement with DSM Biologics places Pharming "in a strong position to further the commercial development of recombinant human C1 inhibitor for Hereditary Angioedema."
Pharming and DSM Biologics have already initiated the technology transfer required for the downstream purification of rhC1INH. The rhC1INH material obtained from DSM will be used to demonstrate equivalence with existing material and may also be used for clinical trials if required.
Dr. Henk Numan, CEO of DSM Biologics regards the deal with Pharming as a way to expand DSM Biologics’ technology portfolio, "to maintain a leading position as Contract Manufacturing Organisation for biopharmaceutical products."
About HAE and and rhC1INH
HAE is a genetic disorder, which is caused by C1 inhibitor deficiency. The disease is characterized by acute attacks of swelling of soft tissues (edema), including regions of the skin, the gut, the mouth and throat. Involvement of the soft tissue of the throat in an attack of angioedema can be fatal. In addition to the life-threatening nature of the disease, quality of life may be seriously impaired.
Currently, treatment of HAE consists of prophylaxis and management of acute attacks. Attacks of angioedema can be effectively treated with intravenous administration of C1 inhibitor purified from human blood. However, C1 inhibitor preparations obtained from human blood have only been approved for use in some European countries. Although blood derived material is shown to be effective for HAE, treatment with such plasma-derived preparations has potential drawbacks in terms of safety and product supply.
Pharming is developing a recombinant version of the human protein that is efficiently produced in milk of animals. After successful completion of a Phase I clinical trial in 2002 in asymptomatic patients, the product is currently being tested in a Phase II clinical trial. Pharming expects to begin phase II/III clinical trials in several European centres later in the year.
Keywords : KW Pharming DSM Biologics Genzyme Flanders Netherlands Contract manufacturing Biomanufacturing Biopharmaceutical Downstream purification Human angioedema HAE Recombinant C1 inhibitor rhC1INH Commercial supply Clinical trial supply material Phase II & III
