Chicago, IL – CSL Behring’s Hemophilia Drug Surpassed by Gene Therapy in Price

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Chicago, IL – The pharmaceutical landscape has reached a new pinnacle in drug pricing as Orchard Therapeutics sets a record with its gene therapy, Lenmeldy™, now the most expensive drug in the U.S. According to recent announcements, Lenmeldy, designed to treat early-onset metachromatic leukodystrophy (MLD), carries a price tag of $4.25 million for a one-time treatment.

Until this development, CSL Behring held the title with its hemophilia B gene therapy, Hemgenix, priced at $3.5 million before discounts. Orchard Therapeutics’ acquisition cost of Lenmeldy reflects the potential life-altering benefits and long-term healthcare savings associated with the treatment of MLD, a rare and fatal genetic disorder.

The price setting takes into account assessments from the Institute for Clinical and Economic Review (ICER), which supports a health-benefit price benchmark up to $3.94 million. Orchard Therapeutics emphasizes that despite the high cost, innovative payment structures and agreements with insurers aim to ensure broad and expedient access to this groundbreaking treatment. Lenmeldy offers hope of halting or slowing the progression of MLD, a condition affecting approximately one in 100,000 live births in the U.S.

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