Orchard’s leukodystrophy gene therapy scores FDA approval
18 Mar 2024
AcquisitionDrug ApprovalGene Therapy
Orchard secured a green light in the US for its metachromatic leukodystrophy (MLD) gene therapy on Monday, adding to several prior European approvals. The FDA clearance also bumps up the value of its acquisition by Japanese firm Kyowa Kirin that closed earlier this year.
Lenmeldy (atidarsagene autotemcel) was approved to treat children with MLD, specifically those who are pre-symptomatic late infantile, pre-symptomatic early juvenile, and early symptomatic early juvenile.
Those with the rare genetic disease are unable to produce the enzyme arylsulfatase A (ARSA), which results in a buildup of sulfatides in cells, damaging the central and peripheral nervous system and causing a loss of motor function, cognitive function, and early death.
Lenmeldy is a one-time gene therapy that comprises autologous CD34+ cells genetically modified to encode the ARSA gene. Patients must undergo a chemotherapy conditioning regimen before receiving the infusion.
Gene therapy boosts survival
The approval was based on data from a pair of clinical trials and an expanded access programme that showed Lenmeldy significantly reduced the risk of severe motor impairment or death, compared with children who didn’t receive the gene therapy.
In total, Lenmeldy was tested in 37 children. All pre-symptomatic late infantile patients reached their sixth birthday; based on natural history data, only about 58% of untreated children with MLD typically survive to six years old.
Additionally, 71% of the gene therapy-treated children at age five were able to walk without assistance, and 85% of all evaluable patients had normal language and performance IQ scores.
Cost-effectiveness pending
Orchard has not yet disclosed a list price for Lenmeldy, but the Institute for Clinical and Economic Review (ICER), a US drug pricing watchdog, said last year that the gene therapy would meet common thresholds for cost-effectiveness if priced between $2.3 million and $3.9 million.
Atidarsagene autotemcel, also known as OTL-200, obtained EU approval as Libmeldy in 2020 as a one-time treatment for early-onset MLD. Libmeldy has also secured reimbursement in the UK, where it has a reported list price of more than £2.8 million ($3.4 million), as well as in Italy, Germany, Sweden, Iceland, Finland and Norway.
Meanwhile, Monday’s approval triggers a bigger payout from Kyowa Kirin, which agreed in October to acquire Orchard for $16 per share, or about $387 million. An additional $1-per-share contingent value right was linked to US approval of the gene therapy, boosting the total buyout to about $478 million.
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