The most expensive gene therapy drug is
Lenmeldy (atidarsagene autotemcel), a one-time treatment for metachromatic leukodystrophy (MLD), with a list price of $4.25 millionper dose. Approved by the FDA in March 2024 for children with early-onset MLD, Lenmeldy is designed to correct the underlying genetic defect that causes this rare and fatal disease, which affects the brain and nervous system. Untreated, many children with the late infantile form of the disease do not survive past age five.
Other high-priced gene therapies include:
- Kebilidi (PTC Therapeutics) for AADC deficiency: $3.95 million per dose.
- Hemgenix (CSL Behring) for hemophilia B: $3.5 million per dose.
- Elevidys (Sarepta Therapeutics) for Duchenne muscular dystrophy: $3.2 million per dose.
- Lyfgenia (bluebird bio) for sickle cell disease: $3.1 million per dose.
- Skysona (bluebird bio) for cerebral adrenoleukodystrophy: $3 million per dose.
- Roctavian (BioMarin) for hemophilia A: $2.9 million per dose.
- Zynteglo (bluebird bio) for beta-thalassemia: $2.8 million per dose.
- Zolgensma (Novartis) for spinal muscular atrophy: $2.32 million per dose.
The high upfront cost of these therapies is often justified by manufacturers based on the significant long-term clinical benefits and the potential to save millions in lifetime healthcare costs associated with chronic management of these rare conditions. Many manufacturers offer outcomes-based agreements to insurers to help manage the initial cost.
