BridgeBio口服infigratinib III期PROPEL 3显示52周生长外获益

Oral Infigratinib Shows Meaningful Benefits Beyond Growth Within 52 Weeks in Children with Achondroplasia in the Phase 3 PROPEL 3 Trial

- Treatment with oral infigratinib for 52 weeks in PROPEL 3 resulted in favorable trends against placebo in clinically meaningful exploratory endpoints including sleep apnea and otitis media events

• Stabilization of sleep apnea measures: The mean total Apnea-Hypopnea Index (AHI) at 52 weeks remained consistent with the baseline mean in the oral infigratinib group, with a 10.4% increase, versus a 49.2% increase in the placebo group; in children younger than 8 years of age, the mean total AHI was unchanged for the oral infigratinib group, versus a 63.2% increase in the placebo group
• Reduction in rate of ear infections: The estimated annualized rate of otitis media events was 38% lower in the oral infigratinib group compared to the placebo group; in children younger than 8 years of age, the annualized rate of otitis media events was 47% lower in the oral infigratinib group compared to the placebo group
  

- In children treated for up to three years in the PROPEL program, oral infigratinib demonstrated sustained improvements in growth (CFBL in height Z-score of +0.92 SD at Year 3) and body proportionality (CFBL in upper-to-lower body segment ratio of -0.15 at Year 3), with a well-tolerated safety profile and no new safety signals

- These findings build on the previously reported PROPEL 3 primary and secondary endpoint results published in NEJM, in which oral infigratinib demonstrated a +2.10 cm/year improvement in AHV versus placebo (p<0.0001) and a statistically significant improvement in body proportionality within 52 weeks in children younger than 8 years of age

- BridgeBio submitted an NDA to the FDA for oral infigratinib in achondroplasia and anticipates a U.S. launch in mid-2027 

PALO ALTO, Calif., Sept. 09, 2026 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage, multi-product biopharmaceutical company focused on developing medicines for genetic conditions, today presented new exploratory analyses from PROPEL 3, the global Phase 3 pivotal study of oral infigratinib in children living with achondroplasia, showing directionally favorable trends beyond growth across medical complications associated with achondroplasia, including sleep apnea, otitis media, and body composition. These data were presented at the Annual European Society for Paediatric Endocrinology (ESPE) Meeting 2026 in Marseille, France, in a late-breaking oral presentation by Julie Hoover-Fong, M.D., Ph.D. of Johns Hopkins University, U.S.

"In the past, achondroplasia research has focused largely on measuring growth because height can be readily determined. But families have consistently emphasized that their priorities extend well beyond the growth chart," said Melita Irving, M.D. of Guy's and St Thomas' NHS Foundation Trust, London, UK. "Growth is only one part of the picture of this medically complicated condition in which children experience disrupted sleep, recurring ear infections, or other health challenges associated with achondroplasia. What I find especially encouraging here is not only the consistent benefit observed across each measure, but that the longer-term data from the PROPEL program show improvements in growth and body proportionality sustained through three years of treatment, and a safety profile that remained favorable with no new safety signals identified. Taken together, these findings suggest there is potential to address a broader range of outcomes that may meaningfully affect a child's health and daily life over time."

The new exploratory results from PROPEL 3 shared at ESPE 2026 include:

• Stabilization of sleep apnea measures:
  • The mean total apnea-hypopnea index (AHI) at 52 weeks remained consistent with the baseline mean in the oral infigratinib group, with a 10.4% increase, versus a 49.2% increase in the placebo group
  • In children younger than 8 years of age, the mean total AHI at 52 weeks was unchanged for the oral infigratinib group, versus a 63.2% increase for the placebo group
• Reduction in rate of ear infections: The estimated annualized rate of otitis media, a recurrent complication in children with achondroplasia that can affect hearing and speech development, was 38% lower in the oral infigratinib group compared to the placebo group, and in children younger than 8 years of age, 47% lower in the oral infigratinib group compared to the placebo group
• Impact on body composition: Mean change from baseline in body mass index was smaller in the oral infigratinib group compared to placebo (0.50 versus 0.93 kg/m²), with a greater increase in lean body mass (1.77 versus 1.58 kg) and smaller increases in body fat mass (0.95 versus 1.12 kg) and visceral fat volume (1.55 versus 18.42 mL) compared to placebo
  

In addition to the late-breaking oral presentation at the Annual ESPE Meeting 2026, BridgeBio shared a poster, Longer-Term Efficacy and Safety Results of Infigratinib in Children with Achondroplasia, presented by Dr. Irving. These findings showed that in children treated for up to three years in the PROPEL program, oral infigratinib demonstrated sustained improvements in growth, with a change from baseline in height Z-score relative to the achondroplasia population of +0.92 SD at Year 3. Additionally, oral infigratinib demonstrated sustained improvements in proportionality, with a change from baseline in upper-to-lower body segment ratio of -0.15 at Year 3. Results showed that oral infigratinib continued to be well-tolerated, with no new safety signals identified.

BridgeBio also shared one poster focused on PROPEL Infant & Toddler (I&T), an ongoing Phase 2/2b study in children under 3 years old with achondroplasia and one eposter focused on qualitative research on the impacts of hypochondroplasia.

PROPEL 3 demonstrated best-in-class improvements in annualized height velocity (AHV) and, for the first time in a Phase 3 achondroplasia study, statistically significant improvements in body proportionality and arm span, supporting its potential as the first oral targeted therapeutic option that directly impacts FGFR3. The topline results can be found here. These data were published as an original research article in the New England Journal of Medicine (NEJM) and simultaneously presented at the International Congress of Children’s Bone Health (ICCBH) 2026 in a late-breaking oral presentation. The results can be found here. 

BridgeBio believes oral infigratinib is positioned to become the first and only approved oral therapy and a potential best-in-class option for children living with achondroplasia. The Company submitted an NDA to the FDA for oral infigratinib in achondroplasia and anticipates a U.S. launch in mid-2027. The Company intends to submit a Marketing Authorization Application (MAA) for achondroplasia to the European Medicines Agency (EMA) in the fourth quarter of 2026. 

Oral infigratinib has received Breakthrough Therapy Designation from the U.S. FDA based on the shared results from the PROPEL 2 clinical trial, which meet the FDA’s requirement of potentially demonstrating substantial improvement in efficacy over available therapies on clinically significant endpoints. In addition to receipt of Breakthrough Therapy Designation, oral infigratinib has also received Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation for achondroplasia from the FDA. If oral infigratinib is approved, BridgeBio may qualify for a Priority Review Voucher. 

Information about PROPEL I&T trial (NCT07169279) can be found here on clinicaltrials.gov. Information about ACCEL, the Company’s observational lead-in study for oral infigratinib in hypochondroplasia’s Phase 3 study (NCT06410976) can be found here, and information about ACCEL 2/3, BridgeBio’s Phase 2/3 clinical study of oral infigratinib in hypochondroplasia (NCT06873035) can be found here. BridgeBio is committed to exploring the potential of oral infigratinib on wider medical and functional impacts of achondroplasia, hypochondroplasia, and other skeletal dysplasia conditions, which hold significant unmet needs for families.