* The British Pharmacological Society awards Cablivi®
(caplacizumab), the first new treatment in 30 years for acquired
Thrombotic Thrombocytopenic Purpura (aTTP), Drug Discovery of the
Year 2020
* TTP is a rare blood clotting disorder with 330 people
known to be affected in England[2]
READING, England, Dec. 17,
2019 /PRNewswire/ -- Sanofi's
Cablivi® (caplacizumab), the first nanobody
therapy for the treatment of acquired Thrombotic Thrombocytopenic
Purpura (aTTP), has been awarded the British Pharmacological
Society's Drug Discovery of the Year 2020 award, at last night's
annual Pharmacology conference.
The Sanofi team were commended by an international judging panel
for the successful translation of caplacizumab from preclinical
development through to approval as a first-in-class nanobody
therapy, which the Society deemed as a pioneering example of the
role of pharmacology in drug discovery and development.
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 (acquired Thrombotic
Thrombocytopenic Purpura), but also as the first-ever nanobody
therapy."
Recognition has also been given by Professor Marie Scully, Consultant Haematologist,
University College London Hospital (UCLH) who commented: "The
discovery of caplacizumab has been a huge and important milestone
for our community. As a rare condition, aTTP is not well known but
once identified, must be diagnosed and treated rapidly.
Caplacizumab's ability to provide rapid protection from potentially
fatal blood clots is vital for both people experiencing aTTP and
the medical teams treating them."
Marc Moodley, Head of Sanofi
Genzyme Medical, commented: "Every day, people at Sanofi work
hard to ensure that our research and development efforts bring
value not only to patients, but to the healthcare landscape as a
whole. To have an independent and prominent UK body recognise this
too, is a true honour. Receiving the Drug Discovery of the Year
2020 award is a fantastic achievement for our team, particularly as
it translates into a real benefit for patients."
About Caplacizumab
Caplacizumab is the first and only specific therapy approved for
the treatment of adults experiencing an episode of aTTP in
conjunction with plasma exchange (PEX) and
immunosuppression.[3] Caplacizumab uses innovative
nanobody technology to provide patients with aTTP rapid
protection from blood clots in small blood
vessels.[3]
Caplacizumab was developed by Ablynx, which was acquired by
Sanofi in 2018. The nanobody technology was originally discovered
following identification that camelidae (e.g. camels and llamas)
possess fully functional antibodies which consist of heavy chains
only and therefore lack light chains.[4] Due to their
small size and unique structure, nanobodies are ideal building
blocks which are small, robust and easily adaptable, resulting in a
targeted biological therapy.[4]
About acquired Thrombotic Thrombocytopenic Purpura
(aTTP)
aTTP is a rare and life-threatening blood clotting disorder
which can result in severe organ damage and
death.[5],[6] It usually presents as a medical emergency
where sudden complications like heart attack and kidney failure can
occur without warning.[6],[7],[8],[9]
About Drug Discovery of the Year
The British Pharmacological Society established the Drug
Discovery of the Year in 2012 to highlight the crucial role
pharmacology plays in the development of new medicines and to
reward the achievements of the teams of scientists who discover new
drugs. The Society's Industry Sub-Committee selected caplacizumab
from a shortlist of excellent first-in-class drugs and outstanding
presentations. To find out more, visit:
https://www.bps.ac.uk/membership-awards/prizes,-awards-and-grants/our-prizes/drug-discovery-of-the-year
About Sanofi UK
Sanofi UK is dedicated to supporting people through their health
challenges. We are a global biopharmaceutical company focused on
human health. We prevent illness with vaccines, provide innovative
treatments to fight pain and ease suffering. We stand by the few
who suffer from rare diseases and the millions with long-term
chronic conditions.
With more than 100,000 people in 100 countries, Sanofi is
transforming scientific innovation into healthcare solutions around
the globe.
Sanofi, Empowering Life
References
[1] The British Pharmacological Society. 2019.
Available
at: https://www.bps.ac.uk/news-events/news/articles/2019/pioneering-treatment-for-life-threatening-blood-cl
Last accessed: December 2019
[2] NHS England. Integrated Impact Assessment
Report for Service Specifications. 2018. Available at:
https://www.engage.england.nhs.uk/consultation/thrombocytopenic-purpura/ user_uploads/thrombotic-thrombocytopenic-purpura-impact-assessment.pdf.
Last accessed: December 2019
[3] EMA. Cablivi Summary of Product
Characteristics. Available at:
https://www.ema.europa.eu/en/documents/product-information/cablivi-epar-product-information_en.pdf.
Last accessed: December 2019
[4] Ablynx. Understanding Nanobodies. Available
at:
https://www.ablynx.com/technology-innovation/understanding-nanobodies/.
Last accessed: December 2019
[5] Knöbl P. Thrombotic thrombocytopenic
purpura. Magazine of European Medical Oncology 2018; 11(3):
220–226
[6] Joly BS, Coppo P, Veyradier. Thrombotic
thrombocytopenic purpura. Blood 2017; 129:2836–2846
[7] Yves Benhamou, et al. Cardiac troponin-I on
diagnosis predicts early death and refractoriness in acquired
thrombotic thrombocytopenic purpura. Journal of Thrombosis and
Haemostasis 2015; 13(2):293–302
[8] Han-Mou Tsai. The kidney in thrombotic
thrombocytopenic purpura. Minerva Medical 2007; 98(6):731–747
[9] Blombery P and Scully M. Management of
thrombotic thrombocytopenic purpura: current perspectives. Journal
of Blood Medicine 2014; 5:15–23