Metachromatic Leukodystrophy (MLD) is a rare, fatal, and recessive genetic disorder caused by a mutation in the ARSA gene, which results in a deficiency of the ARSA enzyme. Without this enzyme, fats accumulate in the brain, nervous system, and other body parts, leading to loss of motor function, cognitive decline, and eventually death.
The disease affects roughly one in every 100,000 births, and approximately 50% of children with the most severe form of MLD die within five years of disease onset.
Lenmeldy is an autologous hematopoietic stem cell-based gene therapy. It was approved in 2024 by the FDA; the European Commission approved it in 2020 (as Libmeldy).
It is suitable for pre-symptomatic or early symptomatic early onset MLD patients. The treatment has proven in clinical trials to extend overall survival and preserve motor and cognitive functions in early-onset MLD patients.
Today, gene therapies, like Lendmeldly, represent a cutting-edge approach in medical science, aiming to treat or prevent diseases by modifying the genetic material within a patient’s cells. Recent regulatory approvals, such as the FDA’s approval of gene therapies for conditions like spinal muscular atrophy and sickle cell disease, highlights the potential of this technology to transform treatment.
Although gene therapy risk for self-funded health plans remains extremely low, it’s important to maintain a strategy for mitigating existing and future treatments. Especially as more gene therapies are approved and become available.
Here we provide a brief summary for today’s approved gene therapies that includes cost estimates and probability for self-funded health plans.
Condition it addresses: Inherited Retinal Disease
Approved by the FDA: December 19, 2017
Claim probability: Estimated that less than 1 claimant per one-million-member plan will receive Luxturna each year
Estimated cost: $425,000 per eye
Condition it addresses: Spinal Muscular Atrophy
Approved by the FDA: May 24, 2018
Claim probability: Estimated that about 1 claimant per one-million-member plan is expected to receive this therapy per year
Estimated cost: $2,255,000
Condition it addresses: Congenital Athymia
Approved by the FDA: October 8, 2021
Claim probability: Most plans will have NO members affected (ultra rare disease state)
Estimated cost: $2,730,000
Condition it addresses: Thalassemia
Approved by the FDA: August 17, 2022
Claim probability: Estimated that no more than one claimant per 1-million-member plan will be treated per year
Estimated cost: $2,800,000
Condition it addresses: Cerebral Adrenoleukody-strophy
Approved by the FDA: September 16, 2022
Claim probability: Most plans will have NO members affected (ultra rare disease state)
Estimated cost: $3,000,000
Condition it addresses: Hemophilia B
Approved by the FDA: November 22, 2022
Claim probability: Estimated up to 1 claimant/year per one-million-member plan to receive due to low incidence/slow uptake
Estimated cost: $3,500,000
Condition it addresses: Bladder Cancer
Approved by the FDA: December 16, 2022
Claim probability: Estimated up to 3 claimants per one-million-member plan to receive each year
Estimated cost: $300,000 per year
Condition it addresses: Dystrophic Epidermolysis Bullosa
Approved by the FDA: May 19, 2023
Claim probability: Estimated that 1 to 2 claimants per one-million-member plan will receive each year
Estimated cost: $630,000/year after induction
Condition it addresses: Duchenne Muscular Dystrophy
Approved by the FDA: June 22, 2023
Claim probability: Estimated up to 1 claimant per one-million-member plan to receive per year
Estimated cost: $3,200,000
Condition it addresses: Hemophilia A
Approved by the FDA: June 29, 2023
Claim probability: Estimated about 1 claimant/year per one-million-member plan to receive (slow initial uptake)
Estimated cost: $2,900,000
Condition it addresses: Sickle cell disease
Approved by the FDA: December 8, 2023
Claim probability: Estimated that in the first 2 years after approval about 1 claimant per 1-million-member plan is expected to receive therapy
Estimated cost: $3,100,000
Condition it addresses: Sickle cell disease AND transfusion dependent beta thalassemia
Approved by the FDA: December 8, 2023; January 16, 2024
Claim probability: For SCD: first 2 years after approval about 1-2 claimants per 1-million-member plan expected to receive therapy; For TDT: 0-1 claimant per 1-million-member plan is expected to receive per year
Estimated cost: $2,200,000
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