UNITED KINGDOM – Orchard Therapeutics announced this week that it has reached an agreement with the NHS in England to cover its gene therapy Libmeldy.

Although Orchard did not specify how much the NHS would pay for the therapy, the agreement is noteworthy given the difficulties gene therapy developers have had in reaching agreements with payers, even when the therapies have been approved.

Bluebird Bio, for example, withdrew all of its gene therapies from Europe last year, claiming that countries were not offering a price commensurate with the value of its drugs for two rare blood disorders and a rare neurological condition.

The European Commission approved Libmeldy in December 2020 for the treatment of children with early-onset metachromatic leukodystrophy, a hereditary condition caused by a gene mutation that renders the body unable to break down certain fats that accumulate in the brain, nerves, and other organs.

According to a recent Lancet publication, 26 of 29 treated patients were still alive, had significant improvements in motor function compared to historical controls, and “most displayed normal cognitive development.”

In Europe, more gene therapies for inherited diseases have been approved than in the United States. However, the track record is not one of commercial success.

Glybera, the first gene therapy approved for use in Europe, was later withdrawn by its developer UniQure after being used only once during its five-year market lifespan.

GlaxoSmithKline developed Strimvelis, a treatment for the genetic condition ADA-SCID, and received European approval for it in 2016. However, few patients were treated because GSK was unable to sell the therapy.

GSK eventually sold Strimvelis and other gene therapy assets to Orchard, which has kept the drug on the market. However, only 16 patients have been treated with it since its approval, owing to the very low incidence of the disease it treats.

Bluebird bio, which received approval for a beta thalassemia gene therapy in 2019 and a rare brain disease treatment last year, recently announced that it would withdraw both medicines from the European market due to reimbursement issues.

Orchard’s goal with Libmeldy is to achieve greater success, and the agreement with the NHS in England and Wales should help.

Despite this, the disease it treats is still extremely rare; an estimated four infants are born with metachromatic leukodystrophy in England each year.

Libmeldy is also only approved for use before symptoms appear or while children retain the ability to walk, emphasizing the importance of early detection.

Orchard is expanding newborn screening through a pilot program in Germany that it hopes to expand to Italy, the United Kingdom, Spain, and France.

In addition to the coverage announcement, Orchard revealed that two patients were treated under early access programs in Germany and France.

After a U.K. agency in charge of health technology assessments had previously recommended against coverage due to concerns about Libmeldy’s long-term effectiveness and cost, the company was able to secure reimbursement.

In a statement, Orchard said it “engaged with the agency to provide additional clinical data and analysis to support the value of Libmeldy and also continued negotiations on the confidential discount.

Libemeldy will be available through the Royal Manchester Children’s Hospital in England, which is one of only five sites in Europe qualified to provide the treatment.

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