BridgeBio’s oral drug improves growth in children with dwarfism in late-stage study

Feb 12 (Reuters) – BridgeBio Pharma said on Thursday its experimental therapy boosted growth rates in children with a rare genetic disorder that causes dwarfism, according to results from a late-stage study.

The oral therapy, infigratinib, improved growth rate by 1.74 cm compared with placebo after 52 weeks in children with achondroplasia, a condition that causes disproportionate short stature.

In a pre-specified analysis of children aged 3 to 8, the therapy also showed statistically significant improvement in body proportions compared with placebo.

BridgeBio said there were no serious side effects related to the drug.

Three cases of high phosphate levels were observed, but all were mild and temporary, and did not require any change in dosing, the company said.

Achondroplasia affects about 55,000 people in the U.S. and European Union, BridgeBio said.

Currently, BioMarin Pharmaceutical’s injectable therapy, Voxzogo, is the only approved drug for the condition.

BridgeBio said it plans to seek regulatory approval in the U.S. and Europe in the second half of 2026. The drug received breakthrough-therapy tag from the U.S. Food and Drug Administration in September 2024.

If approved, infigratinib would be the first oral therapy for achondroplasia, the company said.

The company is also testing the drug in another related condition called hypochondroplasia, a milder form of achondroplasia with less severe growth issues.

(Reporting by Siddhi Mahatole and Sahil Pandey in Bengaluru; Editing by Leroy Leo)