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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 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.

Orchard Therapeutics 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 wasFDAught by Japan’s Kyowa Kirin for $387.4 million in October—has set the whometachromatic leukodystrophy (MLD)e-time gene thKyowa Kirin.25 million, making Orchard Therapeuticspensive drug in the U.S.

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

Lenmeldy’s steep cost is “reflectiCSL Behringalue the theraphemophilia Br … 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.Lenmeldy

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 tLenmeldy centers are planned in Georgia, Pennsylvania, Texas and California.MLD

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. Lenmeldyare genetic disease that targets the MLDin 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 ggenetic diseasenome of a patient’s own hematopoietic stem cells, which are then infused back into the patient.

Lenmeldyrapies like Orchard’s—as bespoke, single-use treatments fARSAiche 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.