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. 


Dr. Oche Otorkpa PG Cert, MPH, PhD

Dr. Oche is a seasoned Public Health specialist who holds a post graduate certificate in Pharmacology and Therapeutics, an MPH, and a PhD both from Texila American University. He is a member of the International Society of Substance Use Professionals and a Fellow of the Royal Society for Public Health in the UK. He authored two books: "The Unseen Terrorist," published by AuthorHouse UK, and "The Night Before I Killed Addiction."
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