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"Debate Over $4.25 Million Gene Therapy for Rare Disease"

1 min read
Source: CNN
"Debate Over $4.25 Million Gene Therapy for Rare Disease"
Photo: CNN
TL;DR Summary

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.

Topics:business#access-to-medicine#expensive-drug#fda-approval#gene-therapy#health#rare-disease
Share this article
  1. A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access  CNN
  2. Kyowa Kirin's gene therapy most expensive US drug with $4.3 million price tag  Yahoo Finance
  3. Kyowa's Orchard hopes $4.25M gene therapy price tag bears fruit  FiercePharma
  4. Orchard Sets $4.25M US Price for Gene Therapy Lenmeldy on Heels of Approval  BioSpace
  5. World's Most Expensive Drug Is Now $4.25 Million Gene Therapy  Bloomberg

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