A pioneering treatment for acute hepatic porphyria – a rare genetic disorder of haem biosynthesis in which toxic molecules build up in the liver - has been named Drug Discovery of the Year 2021. The Alnylam Pharmaceuticals team has been recognised by the British Pharmacological Society for the development of Givosiran (Givlaari™) with its prestigious annual award.
Acute hepatic porphyria attacks are characterised by pain and other signs of neurological dysfunction, often requiring hospitalisation and in severe cases, it can be life-threatening. Givosiran is a small interfering (si)RNA that uses a mechanism of RNA interference to treat hepatic porphyria. It is an si-RNA directed to aminolevulinate synthase 1 (ALAS1) in the liver. By causing the downregulation of ALAS1 mRNA, treatment with Givosiran prevents accumulation of toxins that are associated with acute porphyria attacks.
The Alnylam team were commended by an international judging panel for the development of Givosiran’s novel siRNA mechanism of action to address an area of unmet clinical need. The panel also recognised the important wider potential applicability of the siRNA technology for the treatment of other diseases in the future.
Helen Tomkinson, Chair of the British Pharmacological Society’s Industry Sub Committee, said:
This represents a significant discovery, not just for those with hepatic porphyria The (si)RNA technology used by the Alnylam Pharmaceuticals team could also have an exciting impact on the development of other new medicines.
The Drug Discovery of the Year Award was presented to the Alnylam Pharmaceuticals team by Professor Sir Munir Pirmohamed, President of the British Pharmacological Society, at an online prize-giving ceremony on 18 December 2020, as part of the Society’s annual conference, Pharmacology 2020. The team will also present their work during Pharmacology 2021, taking place from the 7-9th of September 2021.