BridgeBio Publishes Positive Phase 3 Results for Oral Infigratinib in Children with Achondroplasia

BridgeBio Shares Positive Phase 3 Data in NEJM

BridgeBio Pharma has announced that positive results from its global Phase 3 PROPEL 3 study of oral infigratinib in children with achondroplasia have been published in The New England Journal of Medicine (NEJM). The results were also presented during a late-breaking oral presentation at the International Congress of Children's Bone Health (ICCBH) 2026.

The publication marks the first Phase 3 achondroplasia clinical trial to be published in NEJM. It is also BridgeBio's second NEJM publication focused on achondroplasia and its fourth publication in the journal over the past three years.

Phase 3 Study Meets Primary and Secondary Endpoints

The PROPEL 3 trial successfully achieved its primary endpoint by demonstrating a statistically significant improvement in annualized height velocity compared with placebo after 52 weeks of treatment.

Children treated with oral infigratinib experienced an observed mean increase in annualized growth velocity of 2.10 cm per year compared with placebo. The least squares mean treatment difference was 1.74 cm per year, with both results reaching high statistical significance.

According to the company, this represents the largest improvement in annualized growth velocity reported in any Phase 3 achondroplasia study to date.

The study also met its key secondary endpoint by showing a significant improvement in height Z-score compared with baseline after 52 weeks of treatment.

Therapy Shows Improvement in Body Proportionality

One of the most notable findings from the study was the improvement in body proportionality.

In a pre-specified exploratory analysis involving children younger than eight years old, oral infigratinib became the first therapy to demonstrate a statistically significant improvement in body proportionality compared with placebo in a Phase 3 achondroplasia study.

More than half of the participants enrolled in the trial were between three and eight years of age, making this finding particularly important for younger children living with the condition.

New Data Show Better Arm Span Growth

BridgeBio also presented additional data during ICCBH 2026 showing significant improvements in arm span.

Children receiving oral infigratinib showed a statistically significant improvement in arm span compared with placebo, with an increase of 0.37 standard deviations after 52 weeks of treatment.

According to the company, this is the first placebo-controlled achondroplasia trial to report a statistically significant improvement in arm span.

Safety Profile Remains Favorable

The Phase 3 study showed that oral infigratinib was generally well tolerated throughout the trial.

No patients discontinued treatment because of drug-related side effects, and no serious adverse events related to the study medicine were reported.

Only three patients experienced mild, temporary cases of hyperphosphatemia. These events were asymptomatic and did not require dose reductions or treatment discontinuation.

The study also reported no adverse events associated with FGFR1 or FGFR2 inhibition, including retinal or corneal complications.

Additional Research Highlights Daily Impact of Achondroplasia

BridgeBio also presented findings from the observational PROPEL study, which examined the quality of life of children living with achondroplasia.

The research showed that many children experience reduced quality of life, especially in physical functioning, highlighting the daily challenges associated with the condition.

Another study involving children with hypochondroplasia and their families found that commonly used patient-reported outcome questionnaires accurately reflected the real-world physical, cognitive, and quality-of-life challenges experienced by patients. These findings may help support future clinical research and patient care.

Regulatory Filings Planned for 2026

BridgeBio plans to submit a New Drug Application (NDA) for oral infigratinib to the U.S. Food and Drug Administration during the third quarter of 2026.

The company also expects to submit a Marketing Authorization Application (MAA) to the European Medicines Agency in the second half of 2026.

If approved, BridgeBio anticipates launching oral infigratinib in the United States during early to mid-2027.

FDA Designations Support Development

Oral infigratinib has already received several regulatory designations from the U.S. FDA, including Breakthrough Therapy Designation, Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation for achondroplasia.

These designations are intended to support the development and review of treatments for serious conditions with significant unmet medical needs.

About Oral Infigratinib

Oral infigratinib is an investigational small-molecule therapy designed to target the underlying cause of achondroplasia and hypochondroplasia by inhibiting FGFR3 signaling.

Overactive FGFR3 signaling disrupts normal bone growth and leads to disproportionate short stature and other health complications. By reducing this excessive activity, oral infigratinib is designed to improve bone growth at its source.

The therapy is still under clinical development and has not yet received regulatory approval.

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