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First Gene Therapy for Rare Hereditary Neurodegenerative Diseases in Children, Recommended by NICE Draft Guidance | News and Features | News
Gene therapy tidarsageneautotemcel (also known as Libmeldy, created by Orchard Therapeutics) will be available to some children with rare, life-threatening hereditary neurodegenerative states of allogeneic leukodystrophy (MLD). ..
Atidarsagene autotemcel is the first treatment for MLD and the draft guidance considers its use in late infancy or early childhood children. It is believed that MLD gives birth to about five children each year in the United Kingdom.
This one-off treatment, which costs around £ 2.8 million at a list price, is the most expensive drug NICE has ever evaluated. Following NICE’s previous public consultation on draft guidance that did not recommend atidarsagene autotemcel, the company increased secret discounts on drug prices.
Atidarsagene autotemcel is given as a single intravenous infusion. It is designed to correct the genetic cause of MLD by inserting a functional copy of the defective gene into the patient’s own stem cells taken from the bone marrow or blood.
Clinical evidence suggests that tidarsagene autotemcel can improve motor and cognitive function in the short term and correct disease-induced enzyme deficiency.
Cost-effectiveness estimates show that tidarsagene autotemcel provides substantial additional health and quality of life benefits, but these depend on the type of illness.
Atidarsagene autotemcel is also recommended as an option for children in early juvenile conditions who have early clinical symptoms but can still walk independently before the onset of cognitive decline.
Helen Knight, Program director NICE’s Health Technology Evaluation Center said: “The Independent Committee has recognized that MLD is a life-limited, relentless, incapacitated, isolated state that affects all aspects of the lives of patients and caregivers, as well as treatment options for MLD. Recognized that there are important unmet needs for disease-modifying therapies for MLD, which are limited to symptom management.
“The clinical evidence presented shows that for children for whom tidarsagene autotemcel was effective, there is substantial clinical benefit in helping them maintain motor and cognitive function. Therefore, we are pleased that the company has agreed to improve the price discount on the tidarsagene autotemcel, which better reflects the uncertainty of the evidence and the Commission has this potential for children in this state. We are now able to recommend treatments that will change ourselves. “
MLD is caused by a deficiency of the enzyme Arylsulfatase-A. Without this enzyme, a substance called sulfatide accumulates and ultimately destroys the protective myelin sheath of the nervous system. As a result, the cranial and peripheral nerves do not function properly, causing symptoms such as peripheral neuropathy, muscle weakness, sight and hearing loss, difficulty walking, speech loss, cognitive decline, and seizures.
How fast the disease progresses and how long the child lives depends on the age at which the symptoms appear. Children with late-infant MLD (starting 30 months ago, the most common and most rapidly progressing) deteriorate rapidly and usually die between the ages of 5 and 8. Life expectancy is several months to 6 years and 10 to 20 years after onset.
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