The
Following a patient receiving chemotherapy, the autologous hematopoietic stem cell-based gene therapy is administered as a personalised, one-time, single-dose infusion.
The treatment from
About metachromatic leukodystrophy
"Lenmeldy is truly a paradigm-shifting medicine and has the potential to stop or slow the progression of this devastating childhood disease with a single treatment, particularly when administered prior to the onset of symptoms," commented Dr
Deficiency of the enzyme arylsulfatase A (ARSA) and subsequent buildup of sulfatides in the cells is considered to cause MLD, FDA elucidated. This buildup damages the nervous system, resulting in the loss of motor and cognitive function and early death.
Clinical evidence of the gene therapy
FDA stated that the safety and effectiveness of Lenmeldy was assessed based on clinical data, including evidence which showed that Lenmeldy significantly reduced the risk of severe motor impairment or death compared with untreated children.
All the children who were given Lenmeldy were alive at six years of age, compared to only 58 percent of the children not treated with the gene therapy, according to the FDA. Furthermore, "at five years of age, 71 percent of treated children were able to walk without assistance."
The value of Lenmeldy
The figure was determined based on the health benefit price benchmark (HBPB) for Lenmeldy to be up to
The value of Lenmeldy has been recognised by several HTA authorities around the world, including in the US by ICER, which determined Lenmeldy to have the highest value-based price for any treatment it has evaluated to date”
"The value of Lenmeldy has been recognised by several HTA authorities around the world, including in the US by ICER, which determined Lenmeldy to have the highest value-based price for any treatment it has evaluated to date," shared Bennett Smith, Senior Vice President and General Manager of
The gene therapy, known as Libmeldy® in
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