The most expensive drug in the world for the treatment of children is revealed

2024-03-21 14:38:00, Shëndeti CNA
The most expensive drug in the world for the treatment of children is revealed
Illustrative photo

On Monday, the 'American Food and Drug Administration' approved a medicine called 'Lenmeldy', which cures children diagnosed with the rare disease, that of the fatal genetic disorder metachromatic leukodystrophy (MLD).

The price of this drug has been named as the most expensive drug in the world as its price reaches up to 4.25 million dollars. This therapy was approved by the FDA and is the first to treat this devastating disease, which usually kills affected children before they turn 7 years old. About 40 children are born with MLD in the US each year, reports CNN. 

The high price reflects the great benefits of the treatment, but concerns about access and cost for health insurance plans are present. The manufacturing company says it is committed to ensuring broad access for eligible patients.

The price of hope

'Lenmeldy', a new gene therapy for the rare disease 'MLD', uses stem cells from affected people to repair faulty genes. This treatment has been approved by the FDA and offers great long-term benefits for patients. Researchers continue to monitor patients treated with 'Lenmeldy' for more than 12 years and have discovered long-term positive effects. 'MLD' is an inherited disease that causes progressive loss of movement and thinking in children.

The affected families express their hope after the approval of the treatment. One of the affected children from these families benefited from genetic therapy, while their other daughter was successfully treated before symptoms appeared. 

New opportunities for patients

Boston's non-profit Institute for Clinical and Economic Review (ICER) has evaluated the new treatment 'Lenmeldy' for the rare disease 'MLD' and found that its price tag of $4.25 million matches the expected benefits for patients.

However, experts have expressed their concern about the high price of the treatment.

However, they prove that this therapy has a significant impact on affected children, offering them a chance for a normal life. 'ICER' has also evaluated several other genetic therapies, showing an upward trend in the prices of treatments for rare diseases.

Concerns about access and cost of treatment for states and health insurers are growing, emphasizing the need for effective management of health resources./ CNA 

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