Gene Therapies’ Extraordinary Potential: ICER Determines Lenmeldy Treatment for Rare Disease Worth $3.9 Million

The Institute for Clinical and Economic Review, or ICER, is known for its critical stance on the pricing of drugs in the industry. Often referred to as the “nerds” of the drug world, they are seen as bespectacled killjoys who only come out a few times a year to criticize drugmakers for their exorbitant prices. However, last year, ICER stunned many when they determined that a new treatment was worth up to $3.9 million – more than any other medicine in history and equivalent to a 45-year supply of Humira, a commonly used autoimmune drug.

This decision demonstrated the extraordinary potential of a new class of gene therapies to provide genuine cures, something that the pharmaceutical industry rarely accomplishes. The treatment, now approved as Lenmeldy, offers hope to babies born with metachromatic leukodystrophy (MLD), an ultra-rare neurodegenerative disease. Lenmeldy may enable these infants to grow up and lead essentially normal lives, representing a significant breakthrough in medical science.

The approval of Lenmeldy marks a milestone in the gene therapy field and has raised hopes for further advancements in medical treatments. The use of gene therapies has shown promising results in treating genetic diseases that were previously considered untreatable. As such, this decision by ICER highlights the importance of investing in research and development of new treatments that have the potential to save lives and improve quality of life for those affected by rare diseases.