US authorizes first treatment for children with dwarfism

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This authorization “will meet an unmet medical need for more than 10,000 children in the United States,” said in a statement Theresa Kehoe, head at the United States Medicines Agency (FDA).

This treatment, marketed by the BioMarin laboratory under the name Voxzogo, is administered as a subcutaneous injection once a day.

It is authorized for children from the age of five with achondroplasia, the most common form of dwarfism, and who can still grow taller.

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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.

The treatment was tested for a year in a clinical trial on 121 participants between the ages of 5 and 14, some of whom received the BioMarin product, and others a placebo. On average, participants who received the treatment grew 1.57 centimeters taller compared to those who received the placebo, the FDA reported.

The most common side effects were injection site reaction, vomiting, and hypotension.

“More than a decade of scientific research supports the medical breakthrough that Voxzogo represents,” Jean-Jacques Bienaimé, CEO of BioMarin, said in a statement.

The treatment is expected to be available in the United States between the middle and the end of December, according to the company.

It has already been authorized in the European Union since this summer. It’s also under review in Japan, Brazil and Australia, according to BioMarin.

“As a parent of a child with achondroplasia, I see the provision of treatments that impact bone growth as an important step forward,” said Amer Haider, co-founder of the Growing Stronger organization, cited in the BioMarin press release.

During the development of the treatment, some voices were however raised to criticize the possible consequences of such an approach, likely according to them to more stigmatize the dwarf people as “abnormal” and requiring a treatment to cure.

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