The Food and Drug Administration approved the first treatment for the most common cause of dwarfism Friday, a drug that has proved to increase children’s height but has been polarizing among adults with short stature.
The treatment, developed by BioMarin Pharmaceutical, is a once-daily injection for children with achondroplasia, a rare genetic disorder that results in dwarfism and can lead to serious medical complications. In a pivotal clinical trial, patients who got the drug, called Voxzogo, grew 1.6 centimeters more over the course of a year than those who received placebo. That means patients who take Voxzogo throughout childhood are likely to reach heights similar to their peers who don’t have achondroplasia, according to BioMarin.
“It’s the difference between being able to drive a car or not, reaching stuff in closets, being able to take care of your hygiene,” said Jean-Jacques Bienaimé, BioMarin’s CEO. “It would make a huge difference for those patients. There’s no question about it.”
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BioMarin set a list price of about $320,000 per year for the treatment, developed under the generic name vosoritide. Accounting for rebates and discounts, the company expects Voxzogo to bring in about $240,000 in revenue per patient each year. In the U.S., the eligible patient population is about 3,000 people, and Wall Street analysts expect Voxzogo to reach roughly $1 billion a year at its commercial peak. The drug will be available to U.S. patients in December, the company said.
BioMarin does not yet have data on whether Voxzogo can prevent the hearing loss, sleep apnea, and life-threatening skeletal problems that can result from achondroplasia, complications that take years to develop. To some, the company’s focus on height — and the FDA’s willingness to approve drugs based on their ability to make patients taller — threatens to undermine years of advocacy without offering a guaranteed benefit.
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For years, the development of Voxzogo and other investigational treatments for achondroplasia have been fervently debated among people with dwarfism. To thousands of little people, the short stature that comes with achondroplasia is not a disability in need of treatment but a difference to be celebrated.
Organizations like Little People of America, the largest group of its kind in the U.S., have spent years dispelling misconceptions, advocating for fairness, and pointing out that having achondroplasia doesn’t preclude anyone from a fulfilling life. Some members of LPA see drugs like Voxzogo as an existential threat to the community they’ve built. Furthermore, because about 80% of babies with achondroplasia are born to parents of average height, the decision of whether to put a child on Voxzogo will often be made by people unfamiliar with LPA’s work.
LPA has no organizational position on the drug, just as it takes no stance on the limb-lengthening surgeries some little people choose to undergo. Voxzogo’s approval means that, in the future, some members of the group might look different than they do today, but that won’t change LPA’s underlying mission, said Michelle Kraus, the organization’s director of public relations.
“We’ve surveyed the membership in the past, and the most important thing to them is that we provide our service, our support, and our advocacy to people in the dwarfism and short-stature community and their families, no matter what the decisions they make are,” said Kraus.
Voxzogo is approved for children with achondroplasia between the ages of 5 and 18. BioMarin is conducting a study enrolling infants and younger children, with data expected next year. If the results are positive, the company expects to apply for a wider FDA label.
BioMarin’s drug is designed to counteract the genetic mutation that causes achondroplasia. The condition results from an error in a gene called FGFR3, which regulates bone growth. For patients with achondroplasia, the natural process by which cartilage turns into bone is interrupted, resulting in shorter stature, a bowing of the spine, and the risk of serious complications that require surgery.
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