BridgeBio’s Oral Treatment Boosts Growth in Children with Dwarfism in Key Trial
Breakthrough in Treatment for Rare Genetic Disorder
BridgeBio Pharma has announced promising results from a late-stage study of its experimental therapy, infigratinib, which showed significant improvements in growth rates among children with a rare genetic disorder known as achondroplasia. This condition causes disproportionate short stature and affects approximately 55,000 people in the U.S. and European Union.
The oral therapy improved growth rates by 1.74 cm compared to a placebo after 52 weeks in children with achondroplasia. In a pre-specified analysis of children aged 3 to 8, the drug also demonstrated statistically significant improvement in body proportions when compared to a placebo.
BridgeBio reported that there were no serious side effects associated with the drug. However, three cases of high phosphate levels were observed, but all were mild and temporary. These cases did not require any changes in dosing, according to the company.
Currently, BioMarin Pharmaceutical’s injectable therapy, Voxzogo, is the only approved treatment for achondroplasia. BridgeBio plans to seek regulatory approval for infigratinib in the U.S. and Europe during the second half of 2026. The drug received a breakthrough-therapy designation from the U.S. Food and Drug Administration in September 2024.
If approved, infigratinib would be the first oral therapy for achondroplasia, offering a potential alternative to the current injectable treatment. This development could significantly impact the treatment landscape for patients suffering from this condition.
Expanding Research to Other Conditions
In addition to testing inachondroplasia, BridgeBio is also evaluating infigratinib for another related condition called hypochondroplasia. This is a milder form of achondroplasia, characterized by less severe growth issues. By exploring the drug’s efficacy in different conditions, the company aims to broaden its therapeutic applications and provide more options for patients.
Key Points of the Study
- The study focused on children with achondroplasia, a rare genetic disorder causing dwarfism.
- Infigratinib showed a 1.74 cm improvement in growth rate over 52 weeks compared to a placebo.
- Children aged 3 to 8 experienced statistically significant improvements in body proportions.
- No serious side effects were reported, though three cases of high phosphate levels were noted, all of which were mild and temporary.
- The drug is currently under review for regulatory approval in the U.S. and Europe.
- If approved, it would be the first oral therapy for achondroplasia.
Future Prospects
BridgeBio’s ongoing research into infigratinib highlights the potential for innovative treatments in the field of rare genetic disorders. The company’s commitment to expanding the use of this therapy to other conditions like hypochondroplasia underscores its dedication to improving patient outcomes.
With the breakthrough-therapy designation from the FDA and positive results from the late-stage study, infigratinib represents a promising advancement in the treatment of achondroplasia. As the company moves forward with its regulatory submissions, the medical community and patients alike will be watching closely for the next steps in this exciting development.
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