New findings from a trial conducted at Children’s Hospital Colorado and the University of Colorado (CU) Anschutz School of Medicine show that children with achondroplasia, the most common form of dwarfism, have significantly increased growth rates. These results highlight Children’s Colorado’s leadership in pediatric orthopedic research and commitment to advancing treatments for rare skeletal conditions.
Achondroplasia occurs in 1 in 26,000 to 40,000 children and is caused by genetic changes that affect bone growth, proportionality, and differences in the shape of the spine and limbs. Children with this disease are often disproportionately short and may experience health problems involving the spine, legs, ears, nose, and throat.
Children’s Colorado is one of 27 sites in 10 countries participating in this international multicenter study of infigratinib, an investigational oral therapy, sponsored by BridgeBio Pharma. In a randomized, double-blind, placebo-controlled, phase 3 study, 114 children with achondroplasia, ages 3 to <18 years, were treated with either once-daily oral therapy or a placebo for 52 weeks. Children who received oral treatment experienced a statistically significant increase in height, with an observed improvement of 2.10 centimeters per year compared to children who received a placebo. In addition, proportionality improved among children aged 3 to <8 years, the age range in which changes in body proportions are most evident.
“Although growth was the primary endpoint of this study, these findings highlight the broader potential for treatments to improve the overall health and daily lives of children with achondroplasia,” said Rose Brown, MD, MS, Rose Brown Endowed Chair of the Children’s Colorado Orthopedic Institute, professor and associate director of orthopedic surgery at the University of California School of Medicine, and study co-author. “As always, our work is driven by a commitment to making real, meaningful and measurable improvements for the children and families we serve.”
The drug works by targeting the underlying biology of achondroplasia, a condition caused by overactivity of the FGFR3 pathway that inhibits bone growth. As a selective FGFR1-3 inhibitor, oral therapy modulates this signaling and supports bone development.
This research reflects our commitment to improving the lives of children with complex musculoskeletal conditions. We are proud to be part of a global initiative that has the potential to ultimately expand treatment options for patients and families. ”
Dr. Ronald Sokol, Chief Scientific Officer at Children’s Colorado
Researchers say the results are promising, but more time is needed to understand the long-term effects, including how the treatment affects overall health, physical function and quality of life. Most side effects in the study were mild to moderate and similar to common childhood illnesses. No serious side effects or deaths related to the drug have been reported. Participants will continue into the long-term study.
Children’s Colorado is a leading children’s hospital and academic medical center dedicated to improving the health of children through clinical care, research, education and advocacy. Its nationally recognized orthopedic institute provides comprehensive care for children with musculoskeletal conditions, including rare skeletal dysplasias.
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