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Infigratinib

Phase 2

Hypochondroplasia | Small molecule | Rare Disease |BridgeBio Pharma, Inc.|Last Updated: May 26, 2026

Target and mechanism

Molecular targetFGFR3, FGFR1, FGFR4, FGFR2
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedCONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment159

FDA Designations

BREAKTHROUGH_THERAPYORPHAN_DRUGFAST_TRACKRARE_PEDIATRIC_DISEASE

Clinical trial landscape

Infigratinib · 2 trials · 1 indication

Phase 2 2
NCT07393373Open-Label, Long-Term, Extension Study of Infigratinib in Children With HypochondroplasiaHypochondroplasia
ENROLLING BY_INVITATION135 Analytics
NCT06873035An Interventional Study of Infigratinib in Children With HypochondroplasiaHypochondroplasia
ENROLLING BY_INVITATION24 Analytics
PHASE2ENROLLING BY_INVITATION
Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia
HypochondroplasiaUnlock trial analytics
PHASE2ENROLLING BY_INVITATION
An Interventional Study of Infigratinib in Children With Hypochondroplasia
HypochondroplasiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Incidence of treatment emergent adverse events (TEAE) and serious TEAE
10 years
Changes over time in standing height Z-score in relation to HCH and non-HCH growth charts
10 years
Change from baseline (BL) in Annualized Height Velocity (AHV; cm/year)
26 weeks
Incidence, severity, and seriousness of adverse events (AEs) that require dose reduction or discontinuation
at least 26 weeks

Secondary Endpoints

Changes over time in AHV Z-score
10 years
Changes over time in body proportions
10 years
Changes over time in weight Z-score
10 years
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Arm 1: Rollover subjectsEXPERIMENTALChildren who have completed QED-sponsored interventional study with infigratinib
Phase 2 Cohort 1EXPERIMENTALinfigratinib (0.128 mg/kg/day)
Phase 2 Cohort 2EXPERIMENTALinfigratinib (0.25 mg/kg/day)

Interventions

NameTypeDescription
InfigratinibDRUGInfigratinib to be administered by mouth and initiated at the last dose level received in the ACCEL 2/3 study or at the dose selected to be further evaluated after proof-of-concept is established for Phase 2 portion of ACCEL 2/3.
infigratinib 0.128 mg/kg/dayDRUGOral infigratinib 0.128 mg/kg/day
infigratinib 0.25 mg/kg/dayDRUGOral infigratinib 0.25 mg/kg/day
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Eligibility Criteria

Age Range3 Years to 18 Years
SexALL
Healthy VolunteersNo
Study Sites24

Inclusion Criteria: * Inclusion Criteria for Participants Rolling Over from ACCEL 2/3 1. Pediatric participants with HCH who have completed ACCEL 2/3 2. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche Exclusion Criteria: * Exclusion Criteri...

Countries:United StatesAustraliaCanadaFranceNorwayPortugalSingaporeSpainSwedenUnited Kingdom
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Recent Changes (Last 90 Days)

LOWMay 27, 2026NCT07393373startDate: changed
LOWMay 27, 2026NCT07393373startDate: changed
LOWMay 26, 2026NCT06873035primaryCompletionDate: changed
LOWMay 26, 2026NCT07393373primaryCompletionDate: changed
LOWMay 24, 2026NCT06873035studyFirstPostDate: changed
LOWMay 24, 2026NCT07393373studyFirstPostDate: changed

Frequently asked questions about Infigratinib

What is Infigratinib used for?

Infigratinib is an investigational small molecule being studied for the treatment of hypochondroplasia and achondroplasia, two rare skeletal disorders. It is in clinical development for children aged 3 years and older with these conditions. The drug is not yet approved and remains under investigation in clinical trials.

What does Infigratinib target?

Infigratinib is a kinase inhibitor that targets fibroblast growth factor receptors FGFR1, FGFR2, FGFR3, and FGFR4. By inhibiting these receptors, particularly FGFR3, the drug aims to address the underlying signaling abnormalities associated with achondroplasia and hypochondroplasia.

Who is developing Infigratinib?

Infigratinib is being developed by BridgeBio Pharma, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol BBIO. The company is conducting clinical trials to evaluate the drug's safety and efficacy in children with achondroplasia and hypochondroplasia.

What phase is Infigratinib in?

Infigratinib is in Phase 2 clinical development for hypochondroplasia and has completed Phase 2 and Phase 3 trials for achondroplasia. It is an investigational drug and has not received FDA approval. The drug has been granted breakthrough therapy, orphan drug, fast track, and rare pediatric disease designations.

What clinical trials is Infigratinib in?

Infigratinib has been studied in several clinical trials. NCT04265651 and NCT06164951 evaluated the drug in children with achondroplasia, while NCT06873035 is an interventional study in children with hypochondroplasia. NCT07393373 is a long-term extension study in hypochondroplasia. These trials are conducted across multiple countries including the United States, Canada, and European nations.

Is Infigratinib the same as infigratinib 0.128 mg/kg/day?

Yes, infigratinib 0.128 mg/kg/day refers to a specific dosing regimen of infigratinib. The drug is also known by the alternative name infigratinib /kg/day. These names describe the same investigational compound being developed by BridgeBio Pharma for achondroplasia and hypochondroplasia.