Key TakeawaysA new drug boosts growth in children with achondroplasia, a common form of dwarfism.In a clinical trial, children who took the drug grew an average of 1.5 centimeters more than those who did not take the drug.Researchers say it shows promise for the treatment of achondroplasia.

Key Takeaways

A new drug boosts growth in children with achondroplasia, a common form of dwarfism.In a clinical trial, children who took the drug grew an average of 1.5 centimeters more than those who did not take the drug.Researchers say it shows promise for the treatment of achondroplasia.

Results of a phase three trial for a drug to help treat the most common form of dwarfism show it can boost bone growth in children—and sometimes cause them to grow as fast as their peers who don’t have dwarfism.

Results of the trial, which focuses on the drug therapy called vosoritide to treat achondroplasia, were published on September 5 inThe Lancet. The trial was conducted on 121 children at 24 hospitals in seven countries— Australia, Germany, Japan, Spain, Turkey, the U.S., and the U.K.—with patients between the ages of 5 and 17 when they were enrolled.

Study participants received a daily injection of either vosoritide or a placebo for a year. Researchers discovered that children who received vosoritide grew an average of 1.57 centimeters more than those who received the placebo.

That’s a growth rate that’s almost in line with children who don’t have achondroplasia, study co-authorCarlos A. Bacino, MD, a professor of molecular and human genetics at the Baylor College of Medicine and chief of genetics service at the Texas Children’s Hospital, tells Verywell.

While the study results were positive, it’s unclear at this point whether the children who received treatment will be taller as adults or what the long-term effects of the therapy will be, Bacino says.

What This Means For YouIf your child has achondroplasia and you’re concerned about their growth, talk to your doctor about vosoritide. While the drug is not currently FDA approved, it may be possible to enroll in a clinical trial.

What This Means For You

If your child has achondroplasia and you’re concerned about their growth, talk to your doctor about vosoritide. While the drug is not currently FDA approved, it may be possible to enroll in a clinical trial.

What Is Achondroplasia?

Achondroplasia is a bone growth disorder that prevents the changing of cartilage to bone, explains the National Institutes of Health (NIH).People with achondroplasia typically have dwarfism, which results in a limitedrange of motion at the elbows, a large head size, and small fingers.

One in 15,000 to 40,000 newborns worldwide are diagnosed with achondroplasia, according to the NIH.

The Most Common Types of Dwarfism

How Vosaritide Works

Vosoritide blocks the activity of FGFR3 and attempts to give children with achondroplasia normal growth rates. “In a way, it stops or puts the brakes on FGFR3’s inhibitory process,” Bacino says. Children with achondroplasia typically grow around 4 centimeters a year, compared with 6 to 7 centimeters in children without the condition.

Previous trials have shown that the drug is safe to give to people with dwarfism, while this particular trial showed it can be effective at increasing bone growth.

Children who use vosoritide are given daily injections “until the growth plates close, around puberty,” Bacino says. Once a child’s growth plates have closed, he says “you will not have any effect from the drug.”

Achondroplasia is currently treated with monitoring and, in some cases, surgery to address symptoms. But the disease itself isn’t treated.

The condition can cause health complications including:

What Happens Next

The results of the latest trial on vosoritide are promising, but Bacino says that “it’s going to take a few years until we know how well this works over time." He points out that there is some controversy over the use of vosoritide, given that “some families that have achondroplasia may not necessarily consider it a problem.”

“For many families, achondroplasia is an issue,” Bacino says. He cites spinal issues, pain, and neurological issues that can develop as a result of the disease as a reason for treatment.

“We want to prevent some of these complications and allow people with achondroplasia to have a better quality of living,” he says.

2 SourcesVerywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.Savarirayan R, Tofts L, Irving M, Wilcox W, Bacino CA, Hoover-Fong J, et al.Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial.The Lancet. September 5, 2020:396(10252):684-692. doi:10.1016/S0140-6736(20)31541-5National Institutes of Health.Achondroplasia.

2 Sources

Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.Savarirayan R, Tofts L, Irving M, Wilcox W, Bacino CA, Hoover-Fong J, et al.Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial.The Lancet. September 5, 2020:396(10252):684-692. doi:10.1016/S0140-6736(20)31541-5National Institutes of Health.Achondroplasia.

Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.

Savarirayan R, Tofts L, Irving M, Wilcox W, Bacino CA, Hoover-Fong J, et al.Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial.The Lancet. September 5, 2020:396(10252):684-692. doi:10.1016/S0140-6736(20)31541-5National Institutes of Health.Achondroplasia.

Savarirayan R, Tofts L, Irving M, Wilcox W, Bacino CA, Hoover-Fong J, et al.Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial.The Lancet. September 5, 2020:396(10252):684-692. doi:10.1016/S0140-6736(20)31541-5

National Institutes of Health.Achondroplasia.

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