Biotie: BTT1023 receives positive opinion for Orphan Drug Designation

19-Feb-2015 - Finland

The Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending Orphan Drug Designation for Biotie's BTT1023 drug candidate for the treatment of primary sclerosing cholangitis (PSC). PSC is a chronic and progressive fibrotic liver disease for which there are currently no effective therapeutic treatments.

BTT1023 is a fully human monoclonal antibody targeting Vascular Adhesion Protein -1 (VAP-1) and is currently advancing into Phase 2 clinical development. The BUTEO study will be an open label, single arm, multi-centre investigator-sponsored study that will be conducted in partnership with the University of Birmingham, UK and supported through external grant funding. The study is expected to enroll 41 patients and will examine the efficacy, safety and pharmacokinetic properties of BTT1023 in PSC patients. The duration of drug treatment in the study is 11 weeks and the primary efficacy endpoint is reduction of elevated levels of alkaline phosphatase, a blood biomarker of bile duct inflammation. The BUTEO study will be conducted in the UK and is expected to start recruiting patients in Q1/2015.

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Topic world Antibodies

Antibodies are specialized molecules of our immune system that can specifically recognize and neutralize pathogens or foreign substances. Antibody research in biotech and pharma has recognized this natural defense potential and is working intensively to make it therapeutically useful. From monoclonal antibodies used against cancer or autoimmune diseases to antibody-drug conjugates that specifically transport drugs to disease cells - the possibilities are enormous

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Topic world Antibodies

Antibodies are specialized molecules of our immune system that can specifically recognize and neutralize pathogens or foreign substances. Antibody research in biotech and pharma has recognized this natural defense potential and is working intensively to make it therapeutically useful. From monoclonal antibodies used against cancer or autoimmune diseases to antibody-drug conjugates that specifically transport drugs to disease cells - the possibilities are enormous