FDA Approves First Drug for Achondroplasia

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The US Food and Drug Administration (FDA) had approved Voxzogo, the first drug designed to treat achondroplasia.

Achondroplasia is the most common form of dwarfism. The genetic condition results from mutations in the FGFR3 gene that is responsible for bone growth and development. As a result, people develop short and abnormally-shaped bones that give them a short stature. Furthermore, frequent ear infections, sleep apnea, decreased muscle tone, hydrocephalus, and limb shortening can also occur in patients.

There is no known cure for achondroplasia. However, some people respond well to the first year of therapy with human growth hormone. Now, for the first time, researchers have come up with a drug that targets the cause of the condition. In a recent press release, the FDA approved the drug for children aged five years and older with achondroplasia and open epiphyses (growth plates).

With this action, children with short stature due to achondroplasia have a treatment option that targets the underlying cause of their short stature.

Dr. Theresa Kehoe, FDA’s Center for Drug Evaluation and Research

How Does it Work?

The drug, called Voxzogo, was developed by BioMarin Pharmaceutical. The US-based company had been testing the drug for years. Voxzogo binds to natriuretic peptide receptor-B that reduces the gene FGFR3’s activity. Thus, stimulating endochondral bone growth.

This approval is an important milestone representing the first time that physicians will be able to offer a therapy targeted at the root cause of the condition for families of children with achondroplasia aged five and older.

Dr. Lynda Polgreen, trial investigator for Voxzogo

FDA’s approval was based on the result of the drug’s Phase III trials. The year-long, double-blind, placebo-controlled, phase III trial included 121 participants aged 5 years and above. All study participants had achondroplasia with open epiphyses. As part of the study, the participants either received an injection of the drug or a placebo. The researchers then measured the rate of heigh growth the annualized growth velocity of the children. Results of the study showed that the children who received Voxzogo grew an average 1.57cm taller than the placebo group.

Furthermore, the drug most commonly caused reactions at the injection site, vomiting, and decreased blood pressure among the participants. The pharmaceutical company advised adequate hydration and food intake prior to administration for avoidance of low blood pressure. Other adverse reactions included joint pain, diarrhea, fatigue, ear pain, dizziness, and allergy.

Researchers are continuing to assess the drug’s safety and efficacy further, across different ages and over long periods of time.

Source: FDA


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