---
title: "Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia — NEJM report"
id: "pubmed-42370681"
canonical_url: "https://medichelpline.com/clinical-feed/pubmed-42370681"
content_type: "clinical_feed_article"
specialty: "Pediatrics"
source_name: "PubMed / NCBI"
source_url: "https://pubmed.ncbi.nlm.nih.gov/42370681/"
doi: "10.1056/NEJMoa2604565"
published_at: "2026-09-03T00:00:00.000Z"
evidence_level: "Journal Article"
license: "CC-BY-NC-4.0 / Informational Use"
---
# Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia — NEJM report
## Provenance & Clinical Metadata
- **Canonical URL:** https://medichelpline.com/clinical-feed/pubmed-42370681
- **Specialty:** [Pediatrics](https://medichelpline.com/clinical-feed/pediatrics.md)
- **Primary Source:** PubMed / NCBI
- **Source URL:** [Original Journal Publication](https://pubmed.ncbi.nlm.nih.gov/42370681/)
- **DOI:** [10.1056/NEJMoa2604565](https://doi.org/10.1056%2FNEJMoa2604565)
- **Published At:** 2026-09-03T00:00:00.000Z
- **Evidence Rating:** Journal Article
## Executive GIST (TL;DR)
- The PubMed record documents a Phase 3 clinical trial titled "Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia," published in the New England Journal of Medicine (N Engl J Med) in 2026. - The trial is identified as a clinical trial; the PubMed entry includes PMID **42370681** and DOI 10.1056/NEJMoa2604565. - The article lists a large, multi–author international team led by Ravi Savarirayan and including clinicians and researchers from multiple countries and institutions. - Multiple authors are affiliated with pediatric hospitals, university centers, and research institutes across Australia, the United States, Canada, the United Kingdom, France, Spain, Norway, Singapore, Argentina, and other locations. - Industry contributors from BridgeBio Pharma (San Francisco) are included among the authors. - The PubMed page links to the full-text provider (Atypon / NEJM) and reports the e-publication date (Epub 2026 Jun 28) and journal issue (2026 Sep 3;395(9):859-869). - The PubMed entry provides bibliographic metadata (title, authors, affiliations, publication details) but the displayed page does not include the article abstract or trial results in the clipped record provided here. - Specific trial design elements (sample size, inclusion criteria, randomization, dosing, endpoints, efficacy outcomes, safety profile, statistical results) are not reported in the PubMed excerpt shown; readers must consult the NEJM full text or the full PubMed abstract for those details. - The record confirms this is a Phase 3 interventional study of **Oral Infigratinib** in pediatric patients with **achondroplasia**, but further clinical details require access to the primary article or supplemental materials. - Clinicians and researchers seeking trial methods, outcomes, and implications should retrieve the NEJM full text via DOI or institutional access for the complete dataset and analysis.
## Clinical Analysis & Structured Key Points
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Epub 2026 Jun 28. # Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia [Ravi Savarirayan](https://pubmed.ncbi.nlm.nih.gov/?term=Savarirayan+R&cauthor_id=42370681)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-1 "Murdoch Children's Research Institute, Melbourne, VIC, Australia."), [Julie Hoover-Fong](https://pubmed.ncbi.nlm.nih.gov/?term=Hoover-Fong+J&cauthor_id=42370681)[ 2 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-2 "Johns Hopkins University, Baltimore."), [Melita Irving](https://pubmed.ncbi.nlm.nih.gov/?term=Irving+M&cauthor_id=42370681)[ 3 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-3 "Guy's and St. Thomas' NHS Foundation Trust, London."), [Paul Arundel](https://pubmed.ncbi.nlm.nih.gov/?term=Arundel+P&cauthor_id=42370681)[ 4 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-4 "Sheffield Children's NHS Foundation Trust, Sheffield, United Kingdom."), [Josep Maria de Bergua](https://pubmed.ncbi.nlm.nih.gov/?term=de+Bergua+JM&cauthor_id=42370681)[ 5 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-5 "Arthroscopic Surgery Unit, Medical Innovation Knowledge Sharing Hospital, Vitoria-Gasteiz, Spain."), [Philippe M Campeau](https://pubmed.ncbi.nlm.nih.gov/?term=Campeau+PM&cauthor_id=42370681)[ 6 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-6 "Centre Hospitalier Universitaire Sainte-Justine, Montreal."), [Toby Candler](https://pubmed.ncbi.nlm.nih.gov/?term=Candler+T&cauthor_id=42370681)[ 7 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-7 "University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, United Kingdom."), [Benjamin T Cocanougher](https://pubmed.ncbi.nlm.nih.gov/?term=Cocanougher+BT&cauthor_id=42370681)[ 8 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-8 "Division of Human Genetics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati."), [Valerie Cormier-Daire](https://pubmed.ncbi.nlm.nih.gov/?term=Cormier-Daire+V&cauthor_id=42370681)[ 9 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-9 "Hôpital Necker-Enfants Malades, Université Paris Cité, INSERM Unité Mixte de Recherche 1163, Institut Imagine, Paris."), [Thomas Edouard](https://pubmed.ncbi.nlm.nih.gov/?term=Edouard+T&cauthor_id=42370681)[ 10 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-10 "Endocrine, Bone Diseases, and Genetics Unit, Reference Center for Rare Diseases of Calcium and Phosphate Metabolism, Pediatric Research Unit Centre d'Investigation Clinique 1436, Children's Hospital, Toulouse University Hospital, Toulouse, France."), [Svein O Fredwall](https://pubmed.ncbi.nlm.nih.gov/?term=Fredwall+SO&cauthor_id=42370681)[ 11 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-11 "Oslo University Hospital, Oslo.")[ 12 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-12 "Norwegian Center for Rare Diseases, Unit Sunnaas, Nesodden, Norway."), [Paul Harmatz](https://pubmed.ncbi.nlm.nih.gov/?term=Harmatz+P&cauthor_id=42370681)[ 13 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-13 "UCSF Benioff Children's Hospital Oakland, Oakland, CA."), [Daniel Hoernschemeyer](https://pubmed.ncbi.nlm.nih.gov/?term=Hoernschemeyer+D&cauthor_id=42370681)[ 14 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-14 "University of Missouri, Columbia."), [Henrik U Irgens](https://pubmed.ncbi.nlm.nih.gov/?term=Irgens+HU&cauthor_id=42370681)[ 15 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-15 "Haukeland University Hospital, Bergen, Norway."), [Saumya Jamuar](https://pubmed.ncbi.nlm.nih.gov/?term=Jamuar+S&cauthor_id=42370681)[ 16 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-16 "KK Women's and Children's Hospital, Singapore."), [Peter Kannu](https://pubmed.ncbi.nlm.nih.gov/?term=Kannu+P&cauthor_id=42370681)[ 17 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-17 "University of Alberta-Stollery Children's Hospital, Edmonton, Canada."), [Janet M Legare](https://pubmed.ncbi.nlm.nih.gov/?term=Legare+JM&cauthor_id=42370681)[ 18 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-18 "University of Wisconsin School of Medicine and Public Health, Madison."), [Antonio Leiva-Gea](https://pubmed.ncbi.nlm.nih.gov/?term=Leiva-Gea+A&cauthor_id=42370681)[ 19 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-19 "Hospital Universitario Virgen de la Victoria, Instituto de Investigación Biomédica de Málaga, Plataforma Bionand, Hospital Quironsalud Málaga, and Department of Biomedicine, Faculty of Biomedical Sciences and Sports, Universidad Europea de Andalucía, Malaga, Spain."), [Helen McDevitt](https://pubmed.ncbi.nlm.nih.gov/?term=McDevitt+H&cauthor_id=42370681)[ 20 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-20 "NHS Greater Glasgow and Clyde, Glasgow, United Kingdom."), [Roberta Onesimo](https://pubmed.ncbi.nlm.nih.gov/?term=Onesimo+R&cauthor_id=42370681)[ 21 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-21 "Università Cattolica del Sacro Cuore-Fondazione Policlinico A. Gemelli IRCCS, Rome."), [John Phillips 3rd](https://pubmed.ncbi.nlm.nih.gov/?term=Phillips+J+3rd&cauthor_id=42370681)[ 22 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-22 "Vanderbilt Health, Nashville."), [Mariana Del Pino](https://pubmed.ncbi.nlm.nih.gov/?term=Del+Pino+M&cauthor_id=42370681)[ 23 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-23 "Hospital de Pediatría Garrahan, Buenos Aires."), [Marie-Eve Robinson](https://pubmed.ncbi.nlm.nih.gov/?term=Robinson+ME&cauthor_id=42370681)[ 24 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-24 "Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa."), [Massimiliano Rossi](https://pubmed.ncbi.nlm.nih.gov/?term=Rossi+M&cauthor_id=42370681)[ 25 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-25 "Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon, France."), [Mars Skae](https://pubmed.ncbi.nlm.nih.gov/?term=Skae+M&cauthor_id=42370681)[ 26 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-26 "Manchester University NHS Foundation Trust and University of Manchester, Manchester, United Kingdom."), [Leanne M Ward](https://pubmed.ncbi.nlm.nih.gov/?term=Ward+LM&cauthor_id=42370681)[ 27 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-27 "Children's Hospital at London Health Sciences Centre, London, ON, Canada."), [Klane K White](https://pubmed.ncbi.nlm.nih.gov/?term=White+KK&cauthor_id=42370681)[ 28 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-28 "Children's Hospital of Colorado, Aurora."), [Jane Schmidt](https://pubmed.ncbi.nlm.nih.gov/?term=Schmidt+J&cauthor_id=42370681)[ 29 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-29 "BridgeBio Pharma, San Francisco."), [Ted Lystig](https://pubmed.ncbi.nlm.nih.gov/?term=Lystig+T&cauthor_id=42370681)[ 29 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-29 "BridgeBio Pharma, San Francisco."), [Ariana Salvatici](https://pubmed.ncbi.nlm.nih.gov/?term=Salvatici+A&cauthor_id=42370681)[ 29 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-29 "BridgeBio Pharma, San Francisco."), [Yun Bai](https://pubmed.ncbi.nlm.nih.gov/?term=Bai+Y&cauthor_id=42370681)[ 29 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-29 "BridgeBio Pharma, San Francisco."), [Peter W Butler](https://pubmed.ncbi.nlm.nih.gov/?term=Butler+PW&cauthor_id=42370681)[ 29 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-29 "BridgeBio Pharma, San Francisco."), [David van Veenhuyzen](https://pubmed.ncbi.nlm.nih.gov/?term=van+Veenhuyzen+D&cauthor_id=42370681)[ 29 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-29 "BridgeBio Pharma, San Francisco."), [Daniela Rogoff](https://pubmed.ncbi.nlm.nih.gov/?term=Rogoff+D&cauthor_id=42370681)[ 29 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#full-view-affiliation-29 "BridgeBio Pharma, San Francisco.") Affiliations Expand ### Affiliations * 1 Murdoch Children's Research Institute, Melbourne, VIC, Australia. * 2 Johns Hopkins University, Baltimore. * 3 Guy's and St. Thomas' NHS Foundation Trust, London. * 4 Sheffield Children's NHS Foundation Trust, Sheffield, United Kingdom. * 5 Arthroscopic Surgery Unit, Medical Innovation Knowledge Sharing Hospital, Vitoria-Gasteiz, Spain. * 6 Centre Hospitalier Universitaire Sainte-Justine, Montreal. * 7 University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, United Kingdom. * 8 Division of Human Genetics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati. * 9 Hôpital Necker-Enfants Malades, Université Paris Cité, INSERM Unité Mixte de Recherche 1163, Institut Imagine, Paris. * 10 Endocrine, Bone Diseases, and Genetics Unit, Reference Center for Rare Diseases of Calcium and Phosphate Metabolism, Pediatric Research Unit Centre d'Investigation Clinique 1436, Children's Hospital, Toulouse University Hospital, Toulouse, France. * 11 Oslo University Hospital, Oslo. * 12 Norwegian Center for Rare Diseases, Unit Sunnaas, Nesodden, Norway. * 13 UCSF Benioff Children's Hospital Oakland, Oakland, CA. * 14 University of Missouri, Columbia. * 15 Haukeland University Hospital, Bergen, Norway. * 16 KK Women's and Children's Hospital, Singapore. * 17 University of Alberta-Stollery Children's Hospital, Edmonton, Canada. * 18 University of Wisconsin School of Medicine and Public Health, Madison. * 19 Hospital Universitario Virgen de la Victoria, Instituto de Investigación Biomédica de Málaga, Plataforma Bionand, Hospital Quironsalud Málaga, and Department of Biomedicine, Faculty of Biomedical Sciences and Sports, Universidad Europea de Andalucía, Malaga, Spain. * 20 NHS Greater Glasgow and Clyde, Glasgow, United Kingdom. * 21 Università Cattolica del Sacro Cuore-Fondazione Policlinico A. Gemelli IRCCS, Rome. * 22 Vanderbilt Health, Nashville. * 23 Hospital de Pediatría Garrahan, Buenos Aires. * 24 Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa. * 25 Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon, France. * 26 Manchester University NHS Foundation Trust and University of Manchester, Manchester, United Kingdom. * 27 Children's Hospital at London Health Sciences Centre, London, ON, Canada. * 28 Children's Hospital of Colorado, Aurora. * 29 BridgeBio Pharma, San Francisco. * PMID: **42370681** * DOI: [ 10.1056/NEJMoa2604565 ](https://doi.org/10.1056/nejmoa2604565) Item in Clipboard Clinical Trial # Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia Ravi Savarirayan et al. N Engl J Med. 2026. Show details Display options Display options Format Abstract PubMed PMID N Engl J Med Actions * [ Search in PubMed ](https://pubmed.ncbi.nlm.nih.gov/?term=%22N+Engl+J+Med%22%5Bjour%5D&sort=date&sort_order=desc) * [ Search in NLM Catalog ](https://www.ncbi.nlm.nih.gov/nlmcatalog?term=%22N+Engl+J+Med%22%5BTitle+Abbreviation%5D) * [ Add to Search ](https://pubmed.ncbi.nlm.nih.gov/42370681/) . 2026 Sep 3;395(9):859-869. doi: 10.1056/NEJMoa2604565. Epub 2026 Jun 28. ### Authors [Ravi Savarirayan](https://pubmed.ncbi.nlm.nih.gov/?term=Savarirayan+R&cauthor_id=42370681)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#short-view-affiliation-1 "Murdoch Children's Research Institute, Melbourne, VIC, Australia."), [Julie Hoover-Fong](https://pubmed.ncbi.nlm.nih.gov/?term=Hoover-Fong+J&cauthor_id=42370681)[ 2 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#short-view-affiliation-2 "Johns Hopkins University, Baltimore."), [Melita Irving](https://pubmed.ncbi.nlm.nih.gov/?term=Irving+M&cauthor_id=42370681)[ 3 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#short-view-affiliation-3 "Guy's and St. Thomas' NHS Foundation Trust, London."), [Paul Arundel](https://pubmed.ncbi.nlm.nih.gov/?term=Arundel+P&cauthor_id=42370681)[ 4 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#short-view-affiliation-4 "Sheffield Children's NHS Foundation Trust, Sheffield, United Kingdom."), [Josep Maria de Bergua](https://pubmed.ncbi.nlm.nih.gov/?term=de+Bergua+JM&cauthor_id=42370681)[ 5 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#short-view-affiliation-5 "Arthroscopic Surgery Unit, Medical Innovation Knowledge Sharing Hospital, Vitoria-Gasteiz, Spain."), [Philippe M Campeau](https://pubmed.ncbi.nlm.nih.gov/?term=Campeau+PM&cauthor_id=42370681)[ 6 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#short-view-affiliation-6 "Centre Hospitalier Universitaire Sainte-Justine, Montreal."), [Toby Candler](https://pubmed.ncbi.nlm.nih.gov/?term=Candler+T&cauthor_id=42370681)[ 7 ](https://pubmed.ncbi.nlm.nih.gov/42370681/#short-view-affiliation-7 "University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, United Kingdom."), [Benjamin T Cocanougher](https://pubmed.ncbi.nlm.nih.gov/?term=Cocanougher+BT&cauthor_id=42370681)[ 8 ](https://pubmed.ncbi.nlm.nih.gov/4237068
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