Metachromatic Leukodystrophy (MLD)
https://pharmaphorum.com/news/nice-rejects-orchards-gene-therapy-for-rare-childhood-disease-mld/
Orchard Therapeutics’ Libmeldy (OTL-200), a gene therapy for MLD, has been rejected for NHS use by NICE in draft guidance. There is evidence of the short-term benefit of Libmeldy but assumptions about its long-term effects are uncertain, making it unclear whether it will offer value for money to the NHS.
MLD is an inherited neurodegenerative disorder caused by a deficiency in the ARSA gene coding for the enzyme arylsulfatase-A, which allows sulphatide compounds to build up in the body. At high levels, sulphatides become toxic to nerve cells, destroying the insulating myelin sheath that surrounds them and preventing them from working properly. Libmeldy is an ex vivo, autologous, hematopoietic stem cell-based gene therapy – meaning it is based on cells derived from a patient’s own body – and is designed to replace the defective ARSA gene with a working copy. It was approved by the European Commission last December, becoming the first therapy to be registered for early-onset MLD, on the strength of clinical data showing that it could slow down the loss of motor and cognitive function in children with the disease.
The decision kicks off a comment period and further negotiations between the company, NICE and NHS England on the price of Libmeldy which Orchard has already offered a confidential discount on the therapy. The appraisal committee is scheduled to look at Libmeldy again on 6 October.