The United States Food and Drug Administration (FDA) announced that it has approved the first vaccine developed by BioMarin with positive results against achondroplasia, one of the most common causes of dwarfism.
According to the news of Euronews, it was reported that the drug named Voxzogo, which was approved and made available on Friday, was applied daily and increased the height of up to 1.6 centimeters per year in children who were treated compared to the patients who received placebo in clinical tests.
Children receiving treatment may have nearly the same height as their peers.
In his statement, the developer company BioMarin; He noted that it is possible for people with achondroplasia to reach almost the same height as their peers who do not have the disease, provided that they are treated with Voxzogo during childhood. “There is no question that the drug will make a big difference for patients,” said BioMarin General Manager, noting that the main difference is in everyday things such as driving or not, and being able to reach items in lockers.
It was stated that the annual treatment fee of the drug was determined as 320 thousand dollars, and the number of potential buyer patients in the USA was 3 thousand. The company is expected to generate revenue of up to $1 billion annually from this new drug.
No information was shared about whether the Voxzogo treatment developed against achondroplasia provides any protection against life-threatening factors such as hearing loss, sleep apnea and skeletal problems due to the disease.
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