Chemistry:Infigratinib

From HandWiki

Infigratinib is an kinase inhibitor in development for the treatment of achondroplasia and hypochondroplasia.[1][2][3]

Infigratinib is an oral small molecule that targets the fibroblast growth factor receptors FGFR1, FGFR2, and FGFR3.[2][4] Unlike other therapeutics in development or approved for achondroplasia, it is a daily oral capsule.[5][6]

History

Infigratinib is currently in clinical trials for the treatment of children with achondroplasia and hypochondroplasia, conditions caused by variants in the FGFR3 gene.

Recent Phase 3 clinical trial results for infigratinib demonstrated an improvement in annualized growth velocity of +2.1 cm/yr compared to placebo, the largest reported in any Phase 3 trial for achondroplasia.[7][8][9] Infigratinib also demonstrated a statistically significant change in upper-to-lower body proportionality in children 3-8 years of age. There were no treatment-related serious adverse events, and the drug was well-tolerated.[8]

Earlier Phase 2 results of infigratinib for the treatment of achondroplasia were published in the New England Journal of Medicine.[10]

Society and culture

Infigratinib received Breakthrough Therapy Designation from the FDA, the first therapeutic option in development for achondroplasia to receive this designation.[11] Infigratinib was designated an orphan drug by the FDA[12] and the European Medicines Agency in 2021.[13] It was previously approved for medical use for bile duct cancer under the FDA's accelerated approval program in May 2021 at a significantly higher dose than that used for achondroplasia or hypochondroplasia.[14][15]

References

  1. ↑ Cite error: Invalid <ref> tag; no text was provided for refs named Truseltiq APM Summary
  2. ↑ 2.0 2.1 Cite error: Invalid <ref> tag; no text was provided for refs named Truseltiq FDA label
  3. ↑ "Infigratinib Research | FGFR3 & Achondroplasia | QED Tx". BridgeBio. 2026-01-23. https://qedtx.com/our-research/. 
  4. ↑ "Infigratinib (BGJ398): an investigational agent for the treatment of FGFR-altered intrahepatic cholangiocarcinoma". Expert Opinion on Investigational Drugs 30 (4): 309–316. April 2021. doi:10.1080/13543784.2021.1864320. PMID 33307867. 
  5. ↑ "YUVIWEL (Navepegritide)". Drugs@FDA: FDA-Approved Drugs. U.S. Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2026/219164Orig1s000TOC.html. 
  6. ↑ "VOXZOGO (Vosoritide)". Drugs@FDA: FDA-Approved Drugs. U.S. Food and Drug Administration. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=214938. 
  7. ↑ "Once-Weekly Navepegritide in Children With Achondroplasia: The APPROACH Randomized Clinical Trial". JAMA Pediatrics 180 (1). 2026-01-01. doi:10.1001/jamapediatric. ISSN 2168-6203. https://jamanetwork.com/journals/jamapediatrics/fullarticle/2841492. 
  8. ↑ 8.0 8.1 "BridgeBio Reports Positive Phase 3 Topline Results for Oral Infigratinib with the First Statistically Significant Improvements in Body Proportionality in Achondroplasia" (in en-US). BridgeBio Pharma Inc.. 2026-02-12. https://investor.bridgebio.com/news/news-details/2026/BridgeBio-Reports-Positive-Phase-3-Topline-Results-for-Oral-Infigratinib-with-the-First-Statistically-Significant-Improvements-in-Body-Proportionality-in-Achondroplasia/default.aspx. 
  9. ↑ "Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial" (in English). Lancet (London, England) 396 (10252): 684–692. September 2020. doi:10.1016/S0140-6736(20)31541-5. PMID 32891212. 
  10. ↑ "Oral Infigratinib Therapy in Children with Achondroplasia". The New England Journal of Medicine 392 (9): 865–874. February 2025. doi:10.1056/NEJMoa2411790. PMID 39555818. 
  11. ↑ "bridgebio announces infigratinib is the first ever investigational therapeutic option for achondroplasia to be awarded breakthrough therapy designation by the fda" (in en-US). BridgeBio Pharma Inc.. 2024-09-17. https://investor.bridgebio.com/news/news-details/2024/bridgebio-announces-infigratinib-is-the-first-ever-investigational-therapeutic-option-for-achondroplasia-to-be-awarded-breakthrough-therapy-designation-by-the-fda-09-17-2024/default.aspx. 
  12. ↑ "Infigratinib Orphan Drug Designations and Approvals". 11 September 2019. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/listResult.cfm. 
  13. ↑ "EU/3/21/2475". 13 June 2022. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2475. 
  14. ↑ "Drug Trials Snapshots: Truseltiq". 28 May 2021. https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-truseltiq.  Public Domain This article incorporates text from this source, which is in the public domain.
  15. ↑ Center for Drug Evaluation and Research (28 May 2021). "FDA grants accelerated approval to infigratinib for metastatic cholang". FDA. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-infigratinib-metastatic-cholangiocarcinoma.  Public Domain This article incorporates text from this source, which is in the public domain.
  • Clinical trial number NCT02150967 for "A Phase II, Single Arm Study of BGJ398 in Patients With Advanced Cholangiocarcinoma" at ClinicalTrials.gov



Retrieved from "https://handwiki.org/wiki/index.php?title=Chemistry:Infigratinib&oldid=4583152"