In the United States, the Food and Drug Administration (FDA) has approved for the first time a treatment aimed at improving the growth of children with dwarfism. More precisely, this treatment is authorized for children five years and olderwith achondroplasia, the most common form of dwarfism).
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Marketed by the BioMarin laboratory, under the name Voxzogo (vosoritide), it is administered once a day, in the form of a subcutaneous injection.
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More than 10,000 children affected in the United States
Achondroplasia is a genetic disease that affects approximately one in every 15,000 to 40,000 births worldwide. The average height of an adult with achondroplasia is around 1.2 meters. This clearance “will meet an unmet medical need for more than 10,000 children in the United States,” said Theresa Kehoe, director of the general endocrinology division of the FDA’s Center for Drug Evaluation and Research. , in a press release published on November 19 (source 1).
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“People with achondroplasia have a genetic mutation which makes a certain growth-regulating gene (called FGF type 3 receptor) too active, which prevents normal bone growth, the US authorities explain in their press release. Voxzogo works by binding to a specific receptor called the natriuretic peptide receptor B which reduces the activity of the growth regulating gene and stimulates bone growth. “
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More than a decade of scientific research supports the medical advance represented by Voxzogo, insists Jean-Jacques Bienaimé, CEO of BioMarin, in a press release published on November 19 (source 2).
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The treatment was proven for one year in a phase 3 clinical trial involving 121 participants aged 5 and 14 years. Some received the BioMarin product, and others received a placebo. On average, participants who received the treatment grew 1.57 centimeters taller, compared to those who received the placebo., reports the FDA.
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The most common side effects are:
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- a reaction at the injection site (including redness, itching, swelling, bruising, rash, hives, pain),
- vomitings,
- joint pain
- a decrease in blood pressure (hypotension)
- and upset stomach.
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The treatment should be available in the United States by mid-December, says the BioMarin laboratory. It is already authorized in the European Union since August 2021. Marketing authorization reviews are underway in Japan, Brazil and Australia, “with potential approvals in these countries in 2022”, adds BioMarin.
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