All articles tagged with #expensive drug
A new therapy for a rare genetic disease has become the world's most expensive drug, priced at $4.25 million. The treatment targets a rare genetic disorder that causes children to rapidly age and usually results in death during the teenage years. The high cost of the therapy has sparked concerns about accessibility and affordability, raising questions about the pricing of life-saving treatments for rare diseases.
A new gene therapy called Lenmeldy has been approved by the US FDA for the rare and fatal genetic disorder metachromatic leukodystrophy (MLD), with a wholesale price of $4.25 million, making it the world's most expensive medicine. The therapy, which uses stem cells and a harmless virus to insert working copies of a faulty gene, has shown long-term benefits for children with MLD. While the high cost reflects the significant benefits, it raises concerns about access and affordability, especially for public and private health insurance plans. The approval of Lenmeldy represents a significant advancement in treating rare diseases, but its exorbitant price may strain the ability of insurers to cover its costs and limit patient access.