Drugs Q & A

Dwarfs Get Lifeline As FDA Approves New Drug

The FDA has approved the first drug designed to treat short stature caused by achondroplasia, the most common form of dwarfism. 

The agency said Nov. 19 that Voxzogo, made by BioMarin, is designed for kids ages 5 and older with achondroplasia and open epiphyses, or growth plates, which means they still have the potential to grow. The drug targets the underlying cause of short stature caused by achondroplasia. 

Achondroplasia is a genetic condition that causes severely short stature and disproportionate growth. It’s caused by a genetic mutation that causes a certain growth regulation gene to be overly active, preventing normal bone growth. Voxzogo works by binding to a specific receptor that reduces the growth regulation gene’s activity and stimulates bone growth, the FDA said in a news release.  

Voxzogo fulfills an unmet medical need for more than 10,000 kids in the U.S., Theresa Kehoe, MD, director of the FDA’s division of general endocrinology, said in the news release. 

The drug was tested in a yearlong phase 3 trial in which 121 patients were randomly assigned to get either Voxzogo or a placebo. Participants who received the drug grew an average 1.57 centimeters taller compared to those who got the placebo, the FDA said. 

The most common side effects of Voxzogo include injection site reactions, vomiting, and decreased blood pressure. 

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