Orchard sets new gene therapy price tag at $4.25M—the steepest of any drug

2024-03-20

并购基因疗法上市批准

Orchard sets new gene therapy price tag at $4.25M—the steepest of any drug

Preview

来源: FiercePharma

Orchard Therapeutics' price-setting strategy was informed by an assessment from the Institute for Clinical and Economic Review, the U.S. drug cost watchdog, the company said.

After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy (MLD) in the U.S., Kyowa Kirin and its subsidiary Orchard Therapeutics have broken new boundaries in pricing, too.

Orchard—which was bought by Japan’s Kyowa Kirin for $387.4 million in October—has set the wholesale acquisition cost for its one-time gene therapy at $4.25 million, making Lenmeldy the most expensive drug in the U.S.

Until now, that title was held by CSL Behring and uniQure’s hemophilia B gene therapy Hemgenix, which passed muster with U.S. regulators in November 2022 and carries a $3.5 million price tag before discounts.

Lenmeldy’s steep cost is “reflective of the value the therapy may deliver … as well as the potential long-term impact the treatment may have on overall healthcare utilization,” Orchard explained in a press release Wednesday.

Orchard’s price-setting strategy was informed by an assessment from the U.S. drug cost watchdog, the Institute for Clinical and Economic Review (ICER), which determined a health-benefit price benchmark for Lenmeldy at up to $3.94 million.

That is the highest value-based price for any treatment ICER has ever reviewed, Bennett Smith, senior vice president and general manager of North America at Orchard, noted in a statement.

Besides the debate over the med's therapeutic value, affordability could still pose an issue. To that end, the company says it is working in tandem with commercial and government payers to fashion “innovative payment structures” based on outcomes- and value-based agreements meant to reflect the worth of a single, durable treatment.

The deals are "intended to ensure timely access by sharing risk between the payer and manufacturer," the company explained.

Supporting Lenmeldy's launch will be five qualified treatment centers across the U.S. Several eligible kids with MLD have already been treated at one of those centers in Minnesota on a compassionate use basis, Orchard added. That site is in the "final stages of qualification," according to the company.

The other treatment centers are planned in Georgia, Pennsylvania, Texas and California.

Orchard is mapping out its launch strategy just days after Lenmeldy won its FDA approval in early-onset MLD.

MLD affects roughly one in every 40,000 people in the U.S. It’s a rare genetic disease that targets the brain and nervous system, arising from a deficiency of the enzyme arylsulfatase A (ARSA). This ARSA deficit causes a buildup of fatty substances in a person’s cells that deal damage to the central and peripheral nervous systems.

Lenmeldy works by inserting one or more functional copies of the ARSA gene into the genome of a patient’s own hematopoietic stem cells, which are then infused back into the patient.

Gene therapies like Orchard’s—as bespoke, single-use treatments for niche indications—have consistently filled out the top rankings of the most expensive drugs in the U.S. over the past several years.

Beyond Lenmeldy and Hemgenix, there’s also bluebird bio’s Skysona for cerebral adrenoleukodystrophy, which costs $3 million, as well as the biotech’s $2.8 million transfusion-dependent thalassemia drug Zynteglo.