Gene Therapy 101: Meet Lenmeldy™

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).

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.


A NEW GENE THERAPY TO TREAT MLD: LENMELDY 

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.


TOP FACTS TO KNOW ABOUT LENMELDY: 

  • Condition it addresses: Metachromatic Leukodystrophy 
  • Approved by the FDA: March 18, 2024
  • Indications: First FDA-approved treatment for children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile MLD
  • Claim probability: Most health insurance plans will have NO members affected as this is an ultra rare disease state
  • Estimated cost: Projected at $4.25 million plus hospitalization costs.


UNDERSTANDING GENE THERAPY RISK FOR SELF-FUNDED PLANS

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.

Luxturna® 

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

 

Zolgensma®

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

 

Rethymic® 

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

 

Zynteglo® Beta

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

 

Skysona®

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

 

Hemgenix® 

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

 

Adstiladrin® 

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 

 

Vyjuvek®

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

 

Elevidys

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

 

Roctavian 

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

 

Lyfgenia™  

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

 

Casgevy™

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

 

To learn more about how Healthgram can improve your company’s self-funded approach, reach out to a member of our team. 

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