NICE rejects gene therapy for children with neurodegenerative disorder
pharmafile | July 9, 2021 | News story | Medical Communications |
NICE does not recommend gene therapy OTL-200 (also called Libmeldy and made by Orchard Therapeutics) for treating children with the rare inherited neurodegenerative disorder metachromatic leukodystrophy (MLD).
The draft guidance, which is open for public consultation until 30 July 2021, looks at OTL-200 in children with late infantile or early juvenile forms of MLD.
MLD is caused by a deficiency of the enzyme Arylsulfatase-A. Without this enzyme, sulphatides, which are responsible for proper structure and functioning of myelin, build up, eventually destroying the protective myelin sheath of the nervous system. As a result, the nerves in the brain and the peripheral nerves cease to function properly.
Disease progression and life expectancy varies based on age when symptoms appear. Children with the late infantile type of MLD − the most common and the most rapidly progressing − deteriorate rapidly and usually die between the ages of five and eight.
Children with early juvenile MLD have a life expectancy between 10 and 20 years after onset. It is thought there around five children are born each year in England with these types of MLD.
The committee recognised that MLD is a life-limiting, relentless, disabling and isolating condition, affecting all aspects of patients’ and caregivers’ lives. It also recognised that treatment options for MLD are limited to managing symptoms, and that there is a significant unmet need for disease-modifying therapies for MLD.
Although clinical evidence suggests that OTL-200 improves motor and cognitive function and could correct the enzyme deficiency caused by the disease, only short-term clinical evidence is available, so assumptions about long-term stabilisation of symptoms are very uncertain.
The committee therefore concluded that the cost-effectiveness estimates are unlikely to be within the range NICE normally considers an effective use of NHS resources for highly specialised technologies.
The company has proposed a patient access scheme to make OTL-200 available to the NHS with a confidential discount.
Lilly Subbotin
