The Institute for Clinical and Economic Review (ICER) is known as the nerds of the drug industry, often criticizing drugmakers for overpricing their latest cancer and MS treatments. However, last year, ICER staff surprised everyone by valuing a new treatment worth up to $3.9 million, making it the most expensive medicine in history. This valuation was based on the potential of a new class of gene therapies to provide genuine cures for rare and debilitating conditions.
The treatment, now approved as Lenmeldy, has the capability to allow babies born with metachromatic leukodystrophy (MLD), a rare neurodegenerative disease, to live essentially normal lives. This breakthrough demonstrates the power of gene therapies to deliver life-changing treatments for rare and debilitating conditions, offering hope to patients and their families facing these challenges. The approval of Lenmeldy marks a significant milestone in the development of gene therapies as a viable treatment option for genetic diseases.
