A 19-month-old British girl was cured with Liv Meldi, which costs 3 billion won per dose.
The first beneficiary of the world’s most expensive drug has emerged. This is Teddy Shaw, a 19-month-old British girl who was cured with ‘Libmeldy’, a gene therapy method that costs 3 billion won per dose.
On the 15th (local time), The Guardian reported that Teddy Shaw, from Northumberland, England, who was diagnosed with a rare genetic disease, heterochromatic white matter dystrophy (MLD) in April of last year, was treated with Livmelda and was cured.
“It is a moment of great hope for parents and babies born with this deadly genetic disease,” said Amanda Pritchard, Chief Executive Officer of NHS England. can be treated with it,” he said. Teddy’s mother, Allie Shaw, 32, said: “Teddy walks, runs and chats, and is doing just like any other kid.”
Livmeldi (drug name atidarsagene autotemcel) is an MLD treatment developed by Orchard Therapeutics in the UK. MLD is an incurable disease that usually develops in children before 30 months of age and causes vision, speech, and hearing loss, movement disorders, brain disorders, seizures, and eventually death.
Livmeldi was approved for use by the European Medicines Agency (EMA) in October last year, and in January this year, it was approved by the US Food and Drug Administration (FDA) as a treatment called OTL-200. The UK NHS recently signed a special supply agreement with Orchard. That discounted single-capacity price is £2.8 million. Previously, the world’s most expensive drug was ‘Zolgensma’ (drug name: Onasemnoginabeparbovec), a treatment for spinal muscular atrophy (SMA), with a single dose of 2.5 billion won.
MLD is caused by an abnormality in the gene that makes ‘arylsulfatase A’. Arylsulfatase A deficiency causes accumulation of a decomposing enzyme called sulfatide, which damages the myelin sheath (myelin sheath) of the central and peripheral nerves, resulting in organ abnormalities such as peripheral nerves, liver, and kidneys in addition to brain white matter.
Livmeldi corrects the genetic cause of MLD by inserting a functional copy that corrects the defective gene into the patient’s stem cells. Stem cells are extracted from the patient’s own bone marrow or blood and are supplied back into the body with new genetic information. Teddy’s stem cells were removed in several stages from June to October of last year and the defective gene was replaced with a intact gene.
Teddy was lucky. Her older sister Nala, who is three years old, was also diagnosed with MLD last year, but her disease has advanced so much that she cannot benefit from Liv Meldy. Given as a single intravenous infusion, this drug is only effective if given before the irreversible damage caused by the disease has progressed too much.
“When both of my daughters were diagnosed with MLD last April, I thought my world was turned upside down,” Ally Shaw said. “It was most heartbreaking to hear that our first daughter, Nala, would not be able to receive any treatment and would die at a very young age, but we were encouraged by the fact that there was hope for our second daughter, Teddy,” he said. I feel honored and grateful to be the first child.”
About 5 children are born with MLD each year in the UK. Children with MLD onset before 30 months of age rapidly deteriorate and usually die between the ages of 5 and 8 years. If the disease started between the ages of 30 months and 6 years, life expectancy is 10 to 20 years longer.
Live Meldy is provided by the NHS at the Royal Manchester Children’s Hospital in the UK. It is one of five institutions in Europe where Livmeldi can be administered. “We are thrilled to be a part of this landmark moment and to be able to deliver gene therapy that will change the outcome for patients with MLD,” said Professor Rob Winn, director of the hospital’s Pediatric Bone Marrow Transplant Program.
