- The National Health Service in England and Wales will cover treatment with an exceptionally high-priced gene therapy for a rare and fatal genetic disease after negotiating a "significant confidential discount" from the drug's developer, London-based Orchard Therapeutics.
- The therapy, called Libmeldy, was approved by the European Commission in December 2020 for use treating children with early-onset metachromatic leukodystrophy, a hereditary condition caused by a gene mutation that leaves the body unable to break down certain fats that accumulate in the brain, nerves and other organs.
- Securing reimbursement is a significant step for Orchard, which priced Libmeldy at nearly 2.9 million euros per patient treatment. Other gene therapy developers have struggled in the past to win coverage for their medicines in Europe, leading one, Bluebird bio, to recently withdraw two approved products from market.
More gene therapies have been approved for inherited diseases in Europe than in the U.S. But the track record is largely not one of commercial success.
Glybera, the first gene therapy cleared for use in Europe, was later withdrawn by its developer UniQure after being used only once in its five years on the market.
Strimvelis, a treatment for the genetic condition ADA-SCID, was successfully developed by GlaxoSmithKline, which won European approval for it in 2016. But few patients were treated as GSK struggled to sell the therapy.
GSK eventually sold Strimvelis and other gene therapy assets to Orchard, which has continued to market the drug. Only 16 patients, however, have been treated with it since its approval, due as well to the very low incidence of the disease it treats.
More recently, Bluebird bio, which won approval of a beta thalassemia gene therapy in 2019 and a rare brain disease treatment last year, said it would pull both medicines from market in Europe due to difficulties securing reimbursement.
Orchard is aiming for greater success with Libmeldy, and the agreement with the NHS in England and Wales should help. Yet, the disease it treats is still extremely rare; an estimated four infants are born each year in England with metachromatic leukodystrophy. Libmeldy is also only approved for use before symptoms emerge or while children still retain the ability to walk, making early detection important.
"The significance of this milestone likely goes beyond the revenue numbers," wrote Dae Gon Ha, an analyst at Stifel, in a note to clients that described the deal as "a symbolic sign of important progress" for the marketing of gene therapies.
Orchard is working on expanding newborn screening with a pilot program in Germany that it hopes to extend to Italy, the U.K., Spain and France. In addition to its coverage announcement Thursday evening, Orchard also disclosed that two patients were treated under early access schemes in Germany and France.
The company secured reimbursement after a U.K. agency in charge of health technology assessments had earlier recommended against coverage due to questions on Libmeldy's long-term effectiveness and concerns about cost.
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's Children Hospital in England, which is one of five sites in Europe that are qualified to administer the treatment.