Tag: Gene therapy costs

With a price tag of $4.25 million for a single dose, the gene therapy Lenmeldy is the world's most expensive medicine. This revolutionary, one-time treatment addresses metachromatic leukodystrophy (MLD), an ultra-rare genetic disease that is fatal without intervention. The exorbitant cost highlights a new era of ultra-high-priced therapeutics, where new gene therapies continually set new records for what is the most expensive medicine in the world.

In 2019, Novartis's Zolgensma was approved for treating spinal muscular atrophy (SMA) with a record-breaking $2.125 million price tag, quickly earning the reputation as the world's most expensive drug. This one-time gene therapy offered a groundbreaking treatment for a rare and devastating disease. However, in the years since, the field of gene therapy has continued to advance, and Zolgensma's time at the top of the price list has come to an end.