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Infigratinib

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Infigratinib
BERJAYA
Clinical data
Trade namesTruseltiq
Other namesBGJ-398
AHFS/Drugs.comMonograph
MedlinePlusa621041
License data
Pregnancy
category
Routes of
administration		By mouth
Drug classTyrosine kinase inhibitor
ATC code
Legal status
Legal status
Identifiers
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
CompTox Dashboard (EPA)
Chemical and physical data
FormulaC26H31Cl2N7O3
Molar mass560.48 g·mol−1
3D model (JSmol)
  • CCN1CCN(c2ccc(Nc3cc(N(C)C(=O)Nc4c(Cl)c(OC)cc(OC)c4Cl)ncn3)cc2)CC1
  • InChI=1S/C26H31Cl2N7O3/c1-5-34-10-12-35(13-11-34)18-8-6-17(7-9-18)31-21-15-22(30-16-29-21)33(2)26(36)32-25-23(27)19(37-3)14-20(38-4)24(25)28/h6-9,14-16H,5,10-13H2,1-4H3,(H,32,36)(H,29,30,31)
  • Key:QADPYRIHXKWUSV-UHFFFAOYSA-N

Infigratinib is an kinase inhibitor in development for the treatment of achondroplasia and hypochondroplasia.[1][4][5]

Infigratinib is an oral small molecule that targets the fibroblast growth factor receptors FGFR1, FGFR2, and FGFR3.[4][6] Unlike other therapeutics in development or approved for achondroplasia, it is a daily oral capsule.[7][8]

History

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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.[9][10][11] 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.[10]

Earlier Phase 2 results of infigratinib for the treatment of achondroplasia were published in the New England Journal of Medicine.[12]

Society and culture

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Infigratinib received Breakthrough Therapy Designation from the FDA, the first therapeutic option in development for achondroplasia to receive this designation.[13] Infigratinib was designated an orphan drug by the FDA[14] and the European Medicines Agency in 2021.[15] 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.[16][17]

References

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  1. 1 2 3 "Truseltiq". Therapeutic Goods Administration (TGA). 22 November 2021. Retrieved 28 December 2021.
  2. ↑ "Updates to the Prescribing Medicines in Pregnancy database". Therapeutic Goods Administration (TGA). 12 May 2022. Archived from the original on 3 April 2022. Retrieved 13 May 2022.
  3. ↑ "Summary Basis of Decision (SBD) for Truseltiq". Health Canada. 23 October 2014. Archived from the original on 29 May 2022. Retrieved 29 May 2022.
  4. 1 2 3 "Truseltiq- infigratinib capsule". DailyMed. Archived from the original on 10 June 2021. Retrieved 10 June 2021.
  5. ↑ "Infigratinib Research | FGFR3 & Achondroplasia | QED Tx". BridgeBio. BridgeBio. 23 January 2026. Retrieved 15 August 2024.
  6. ↑ Botrus G, Raman P, Oliver T, Bekaii-Saab T (April 2021). "Infigratinib (BGJ398): an investigational agent for the treatment of FGFR-altered intrahepatic cholangiocarcinoma". Expert Opinion on Investigational Drugs. 30 (4): 309–316. doi:10.1080/13543784.2021.1864320. PMID 33307867. S2CID 229177726.
  7. ↑ "YUVIWEL (Navepegritide)". Drugs@FDA: FDA-Approved Drugs. U.S. Food and Drug Administration. Retrieved 16 June 2026.
  8. ↑ "VOXZOGO (Vosoritide)". Drugs@FDA: FDA-Approved Drugs. U.S. Food and Drug Administration. Retrieved 16 June 2026.
  9. ↑ Savarirayan R, McDonnell C, Bacino CA, Hoernschemeyer DG, Legare JM, Abuzzahab MJ, et al. (1 January 2026). "Once-Weekly Navepegritide in Children With Achondroplasia: The APPROACH Randomized Clinical Trial". JAMA Pediatrics. 180 (1). doi:10.1001/jamapediatric. ISSN 2168-6203. Archived from the original on 18 May 2026.
  10. 1 2 "BridgeBio Reports Positive Phase 3 Topline Results for Oral Infigratinib with the First Statistically Significant Improvements in Body Proportionality in Achondroplasia". investor.bridgebio.com. BridgeBio Pharma Inc. 12 February 2026. Retrieved 16 June 2026.
  11. ↑ Savarirayan R, Tofts L, Irving M, Wilcox W, Bacino CA, Hoover-Fong J, et al. (September 2020). "Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial". Lancet. 396 (10252). London, England: 684–692. doi:10.1016/S0140-6736(20)31541-5. PMID 32891212.
  12. ↑ Savarirayan R, De Bergua JM, Arundel P, Salles JP, Saraff V, Delgado B, et al. (February 2025). "Oral Infigratinib Therapy in Children with Achondroplasia". The New England Journal of Medicine. 392 (9): 865–874. doi:10.1056/NEJMoa2411790. PMID 39555818.
  13. ↑ "bridgebio announces infigratinib is the first ever investigational therapeutic option for achondroplasia to be awarded breakthrough therapy designation by the fda". investor.bridgebio.com. BridgeBio Pharma Inc. 17 September 2024. Retrieved 16 June 2026.
  14. ↑ "Infigratinib Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 11 September 2019. Archived from the original on 28 October 2022. Retrieved 30 May 2021.
  15. ↑ "EU/3/21/2475". European Medicines Agency. 13 June 2022. Archived from the original on 30 May 2023. Retrieved 1 August 2023.
  16. ↑ "Drug Trials Snapshots: Truseltiq". U.S. Food and Drug Administration (FDA). 28 May 2021. Archived from the original on 28 July 2023. Retrieved 1 August 2023. Public Domain This article incorporates text from this source, which is in the public domain.
  17. ↑ Center for Drug Evaluation and Research (28 May 2021). "FDA grants accelerated approval to infigratinib for metastatic cholang". U.S. Food and Drug Administration. FDA. Archived from the original on 2 August 2023. Retrieved 1 August 2023. Public Domain This article incorporates text from this source, which is in the public domain.
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  • Clinical trial number NCT02150967 for "A Phase II, Single Arm Study of BGJ398 in Patients With Advanced Cholangiocarcinoma" at ClinicalTrials.gov
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