July 13, 2024

Medical Trend

Medical News and Medical Resources

World Most Expensive Drug Sets New Record: $4.25 Million Per Dose

World Most Expensive Drug Sets New Record: $4.25 Million Per Dose

World Most Expensive Drug Sets New Record: $4.25 Million Per Dose

Recently, Orchard Therapeutics’ new drug, Lenmeldy (atidarsagene autotemcel, formerly known as OTL-200), received approval from the U.S. FDA for treating metachromatic leukodystrophy (MLD). The wholesale cost for a single treatment in the United States is $4.25 million, setting a new record for the most expensive drug.

World Most Expensive Drug Sets New Record: $4.25 Million Per Dose

Developed by the American pharmaceutical company Orchard Therapeutics, Lenmeldy was approved by the FDA on March 18 for treating metachromatic leukodystrophy (MLD), a rare and fatal genetic disorder.

MLD is a rare disease caused by a genetic mutation. Patients lack a critical enzyme called ARSA, leading to progressive damage to the nervous system and developmental decline. Children with MLD face severe developmental challenges and life-threatening conditions.

One of the most immediate and dangerous threats is the continuous production of phlegm. Even healthy infants occasionally choke due to uncoordinated muscle use. For children with MLD, impaired neural development makes it extremely difficult to coordinate the muscles controlling swallowing. A small amount of phlegm, which might be a minor discomfort for others, can cause severe respiratory issues or even threaten their lives.

In addition to phlegm, children with MLD suffer from pain, frequent infections, and seizures, significantly impacting their quality of life and accelerating the disease’s progression. Most affected children die within five years of onset, making MLD a critical challenge for the medical community.

However, the introduction of Lenmeldy brings hope to these children.

How Effective is Lenmeldy?

Lenmeldy is a gene therapy using hematopoietic stem cells (HSCs). It involves genetically modifying the patient’s HSCs outside the body and reintroducing them. Some modified cells enter the central nervous system and express the functional enzyme, fundamentally correcting the genetic cause of MLD.

According to the FDA, Lenmeldy’s safety and efficacy are based on data from 37 children. The primary efficacy endpoint was “survival without severe motor disability,” defined as the time from birth to the first occurrence of losing the ability to sit unassisted or death.

The study results showed that Lenmeldy significantly reduced the risk of severe motor disability or death in children with MLD compared to untreated children. All treated pre-symptomatic late-infantile MLD children survived to age six, whereas the natural survival rate at age six for untreated children is only 58%.

World Most Expensive Drug Sets New Record: $4.25 Million Per Dose

At age five, 71% of treated children could walk unassisted. Additionally, 85% of treated children had normal language and performance IQ scores, while untreated children did not. For children with pre-symptomatic juvenile or early-symptomatic early juvenile MLD, treatment slowed the progression of motor and cognitive impairments.

Why is Lenmeldy So Expensive?

The U.S. Institute for Clinical and Economic Review (ICER) estimates that if Lenmeldy’s price is between $2.3 to $3.9 million, it would be cost-effective, making the current high price reasonable.

Lenmeldy is a one-time, individualized, single-dose infusion made from the patient’s own hematopoietic stem cells. Doctors genetically modify these cells outside the body to produce the missing ARSA enzyme, then reinfuse them into the patient. Some modified cells cross the blood-brain barrier and express the required enzyme in the brain.

This innovative treatment method fundamentally corrects the genetic defect of MLD to some extent, bringing new hope and life to affected children.

World Most Expensive Drug Sets New Record: $4.25 Million Per Dose

Image source: Orchard gene therapy brochure

Not only is Lenmeldy expensive, but other gene therapies also have high costs due to their nature. Currently, 90% of approved gene therapies are for rare diseases, with about 80% caused by genetic defects. In 2023, the FDA approved five gene therapies, four of which target rare diseases: Casgevy and Lyfgenia for sickle cell disease, Roctavian for hemophilia A, and Elevidys for Duchenne muscular dystrophy.

The rare disease market is small, and the necessity for customized treatments for each patient inevitably leads to high prices.

World Most Expensive Drug Sets New Record: $4.25 Million Per Dose

(source:internet, reference only)

Disclaimer of medicaltrend.org

Important Note: The information provided is for informational purposes only and should not be considered as medical advice.