The US health authorities have announced that they have approved, for the first time, a treatment that could improve the development of children suffering from dwarfism.
The drug approval “will cover a medical need for more than 10,000 children in the United States,” said Teriza Kiho, a senior FDA official.
BioMarin Voxzogo is injected subcutaneously once a day. Recommended for children 5 years and older who suffer from achondroplasia, the most common cause of dwarfism, who could be taller.
Achondroplasia is a genetic disease that affects approximately 1 child per 15,000-40,000 births worldwide. On average, adults reach a height of 1.2 meters.
The treatment was tested in a clinical trial involving 121 minors aged 5-14 years.
Some took BioMarin and others took placebo. On average, those who took the drug grew 1.57 cm taller than those who took the placebo.
The most common side effects of the drug were injection site skin reaction, vomiting and hypotension.
The drug has already been approved in the European Union since the summer. It is also under consideration in Japan, Brazil and Australia, according to BioMarin.
“As a parent of a child with achondroplasia, I believe that providing bone growth therapies is an important step forward,” said Amer Haider, co-founder of Growing Stronger.
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