The first nanobody therapy to treat adults with acquired thrombotic thrombocytopenic purpura (aTTP), a rare life-threatening autoimmune blood-clotting disorder, has been named Drug Discovery of the Year 2020. The team of Belgian researchers from Sanofi has been recognised by the British Pharmacological Society for the development of Caplacizumab (Cablivi®) with its prestigious annual award. Caplacizumab was developed by Ablynx, which was acquired by Sanofi in 2018.
aTTP is characterised by extensive clot formation in small blood vessels throughout the body, leading to severe thrombocytopenia (very low platelet count), microangiopathic hemolytic anemia (loss of red blood cells through destruction), ischemia (restricted blood supply to parts of the body) and widespread organ damage especially in the brain and heart.
Caplacizumab is a bivalent single domain antibody fragment that blocks the interaction of ultralarge von Willebrand Factor (vWF) multimers on platelets. It therefore has an immediate effect on platelet adhesion, inhibiting the formation and accumulation of the micro-clots that cause the severe thrombocytopenia, tissue ischemia and organ dysfunction.
The Sanofi team were commended by an international judging panel for the successful translation of Caplacizumab from preclinical development to clinical studies and approval as a first-in-class nanobody therapy. Their success is a pioneering example of the role of pharmacology in the drug discovery and development process.
Dr Filip Callewaert, Medical Affairs Director for Cablivi® in Europe, said:
Given the quality of the medicines that have received this prestigious award, the team is delighted that its work on Caplacizumab has been recognised by the Committee as the British Pharmacological Society’s Drug Discovery of the Year 2020. Caplacizumab is the result of a truly cross-functional collaboration and represents a much-needed therapeutic advance for patients suffering from aTTP.
Dr Tom Blackburn, Chair of the British Pharmacological Society’s Industry Sub-Committee, said:
Caplacizumab represents a major innovation, not only in treating this life-threatening disorder, but also as the first-ever nanobody therapy.
The Drug Discovery of the Year Award was presented to the Sanofi team by Professor Steve Hill, President of the British Pharmacological Society, at a prize-giving ceremony on 16 December 2019 as part of the Society’s annual conference, Pharmacology 2019.