Arexis obtains orphan drug designation in the EU for novel cystic fibrosis therapy

GOTHENBURG, SWEDEN –8 February 2005 – The Gothenburg-based drug development company Arexis AB today announced that the European Commission has granted orphan drug status for BSSL, the novel human enzyme replacement therapy for treatment of fat malabsorption in patients with cystic fibrosis. ”The orphan drug designation further underlines the positive results in our clinical trials and supports Arexis’ development towards becoming an integrated pharmaceutical company”, says Dr. Lennart Hansson, CEO at Arexis.

In September 2004, Arexis presented successful results from its Phase II clinical trial on enzyme replacement therapy in cystic fibrosis. The study showed a more rapid and fully restored lipid uptake for cystic fibrosis patients when supplemented with the enzyme BSSL (bile salt-stimulated lipase), compared to conventional treatments using pancreas extract from pigs. The European Committee for Orphan Medicinal Products (COMP) ruled that recombinant human BSSL satisfies the criteria for orphan drug status in the European Union. COMP asserts that even though satisfactory methods of treatment of cystic fibrosis have been authorised, justifications have been provided that the recombinant human BSSL may be of significant benefit to those affected by the condition. The regulatory authorities in the EU and the US grant orphan drug status to novel, effective treatments in therapy areas with relatively small patient populations. An orphan drug designation grants the holder market exclusivity for up to 10 and 7 years, respectively. According to the report issued by EMEA (European Medicines Agency), cystic fibrosis affects about 1.3 in 10,000 people in the EU. An orphan drug application has been submitted in the US with the objective to obtain orphan drug designation in 2005. Arexis is planning a dose-finding study and a phase III trial with protocol assistance from regulatory authorities in order to obtain market authorisation approval. The total global market of the enzyme replacement therapy segment is estimated to exceed US$ 1 billion.