FDA approves innovative gene therapy for MLD

The US Food and Drug Administration (FDA) has approved gene therapy Lenmeldy ™ (atidarsagene autotemcel) as the first treatment for children with early-onset metachromatic leukodystrophy (MLD).

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 Orchard Therapeutics, a company recently acquired by Kyowa Kirin, is indicated for children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile MLD.

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 Bobby Gaspar, PhD, co-founder and CEO of Orchard Therapeutics.

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

Orchard Therapeutics stated that the wholesale acquisition cost (WAC) of Lenmeldy in the US is $4.25 million.

The figure was determined based on the health benefit price benchmark (HBPB) for Lenmeldy to be up to $3.94 million at the $150,000 per Equal Value Life Year (evLY) threshold from a modified societal perspective, according to the company.

“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 North America at Orchard Therapeutics.

The gene therapy, known as Libmeldy^® in Europe, has already been approved by the European Commission, UK Medicines and Healthcare products Regulatory Agency (MHRA), and Swiss Agency for Therapeutic Products (Swissmedic).