Swedish Orphan Biovitrum announced that the European Commission has granted orphan drug designation to its long-lasting, fully-recombinant Factor FVIII Fc fusion protein (rFVIIIFc), which is partnered with Biogen Idec. It was recently announced that the companies plan to advance the rFVIIIFc programme into a registrational clinical trial in patients with severe haemophilia A.
Treatment of severe haemophilia A requires frequent infusions, creating a significant burden for individuals with the condition. The rFVIIIFc molecule, which is based on Biogen Idec's novel and proprietary monomeric Fc-fusion technology, is being investigated for the potential to prolong protection from bleeding and reduce the frequency of injections for both prophylaxis and on-demand therapy in haemophilia A.
"The orphan drug designation is very valuable for our promising and high-opportunity rFVIIIFc project as it allows European Medicine Agency fee reduction, protocol scientific advice, and gives market exclusivity once the product is approved and receives orphan status. This will help us in our efforts to bring this innovative rFVIIIFc product to haemophilia patients," said Peter Edman, Ph.D., chief scientific officer of Swedish Orphan Biovitrum.
Haemophilia A is a rare, inherited disorder in which the ability of a person's blood to clot is impaired. Haemophilia A occurs in about 1 in 10,000 male births annually and is caused by having substantially reduced or no factor VIII protein, which is needed for normal blood clotting. People with haemophilia A therefore need injections of factor VIII to restore the coagulation process and prevent frequent bleeds that could otherwise lead to pain, irreversible joint damage and life-threatening haemorrhages. Prophylaxis treatment with infusions three times per week or every second day to maintain a sufficient circulating level of coagulation factor is being increasingly used, and long-term studies demonstrate that such regimens increase the patient's life expectancy and greatly reduce if not eliminate progressive joint deterioration. The current global market for recombinant Factor VIII products exceeds 4 BUSD annually.
Swedish Orphan Biovitrum is a Swedish based niche specialty pharmaceutical company with an international market presence. The company is focused on providing and developing specialist pharmaceuticals for rare disease patients with high medical needs.