ShareRefrigeration at the heart of biomedical innovation: the example of Libmeldy
A rare inherited disorder can now be cured with gene therapy. Libmeldy, an innovative drug, must be stored at a temperature of – 130°C.
Metachromatic leukodystrophy is a rare hereditary disease characterised by a change in a gene required for the production of the enzyme ARSA. This enzyme enables the breakdown of certain substances known as sulfatides. When not broken down, sulfatides damage organs and the nervous system.
After more than ten years of research, a drug was recently launched on the market. To date, 11 European countries have decided to reimburse this extremely expensive drug: with a dose costing USD 4.25 million, Libmeldy is now the most expensive drug in the world. This drug is also known as “Lenmeldy” in the US.
Libmeldy is a gene therapy: one or more functional copies of the diseased gene are inserted. They are attached to a harmless retrovirus that transports copies of the healthy gene. The genetically modified cells are then injected into the patient. This cutting-edge technology explains the cost of this drug.
This treatment also requires very strict storage conditions: Libmeldy's infusion bags must be kept in the vapour phase of liquid nitrogen at – 130°C until they are thawed and administered.
Very low temperatures are increasingly being used to preserve heat-sensitive healthcare products. The global market for temperature-sensitive healthcare products, preserved at controlled temperatures, has expanded considerably over the last decade and is expected to continue to grow. The best-known example is, of course, the Pfizer-BioNTech vaccines (stored at temperatures of –80°C to –60°C), Moderna (–25°C to –15°C) and Johnson & Johnson (2°C to 8°C) marketed during the COVID-19 epidemic.
Did you know?
In 2021, the International Institute of Refrigeration and UNEP co-edited a cold chain technology brief dedicated to vaccines. It is available here: https://iifiir.org/en/fridoc/cold-chain-technology-brief-vaccines-144036
Sources:
https://ela-asso.com/actualites/remboursement-libmeldy/ (in French)