Orchard Therapeutics groundbreaking stem cell gene therapy Libmeldy has proven to be effective against metachromatic leukodystrophy disease (MLD), a rare and fatal genetic disorder with no other known cures.

Libmeldy scoredapprovalinthe European Union lastDecemberand a month later nabbeda regenerative medicine advanced therapy (RMAT) designation by the FDA to speed its path to approval in the United States.

But according to Englands drug price watchdog, Libmeldy is too expensive and has yet to be proven over the long term.

In draft guidance, the National Institute for Health and Care Excellencerejectedthe gene therapy for use in the U.K.

On an earnings call last August, Orchard CEO Bobby Gaspar said he expected Libmeldy to carry a price between 2.5 and 3 million ($3-3.5 million) and provided a rationale for the cost.

The price is "less than the average 10-year cumulative cost for many chronic or lifelong rare disease therapies such as certain enzyme replacement therapies on the market today, which do not offer the potential for full genetic correction or a potentially positive impact on cognitive outcomes, Gaspar said during the call.

"We are open to flexible payment arrangements, including value-based payment options over five years or more," Gaspar added.

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The first patient to undergo the therapy got their dose morethan a decade ago. So far, researchers have collectedeight years of follow-up data from the first six patients to undergo the treatment. The drug has proven to preserve motor and cognitive function, Orchard says.

NICEs decision is not final as the agency has established a discussion period through July 30. A third evaluation committee meeting is set for October.

During the process, London-based Orchard will have the opportunity to adjust Libmeldys price. The company hasalreadyoffereda confidential discount.

MLD strikes roughly five children per year born in the U.K. It occurs in roughly 1 in every 150,000 live births.

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MLD is caused by mutations in the ARSA gene, which lead to neurological damage and development regression. The disorders effects are similar to those with multiple sclerosis, but progress more rapidly. In the early-onset form of the disease, young children lose the ability to walk, talk and interact. Most die before adolescence. The disease also can strike older children.

The treatment is created by using a patients own stem cells. During production, the company inserts copies of the ARSA geneinto the genome of the stem cells using a lentiviral vector. Then, the genetically modified cells are infused back into the patient. The gene-corrected stem cells have the ability to correct the underlying disease with a single treatment.

Read more:

England's drug price watchdog rejects Orchard's groundbreaking gene therapy after first round of talks - FiercePharma

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