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Infigratinib/kg

Phase 3

Achondroplasia | Small molecule | Rare Disease |BridgeBio Pharma, Inc.|Last Updated: Mar 17, 2026

Target and mechanism

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

Also known as Infigratinib

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials3
Total Enrollment498

FDA Designations

BREAKTHROUGH_THERAPYORPHAN_DRUGFAST_TRACKRARE_PEDIATRIC_DISEASE

Clinical trial landscape

Infigratinib/kg · 3 trials · 1 indication

Phase 3 1Phase 2 2
NCT06164951A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With AchondroplasiaAchondroplasia
COMPLETED114 Analytics
PHASE3COMPLETED
A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia
AchondroplasiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Change from baseline (BL) in annualized height velocity (cm/year)
Week 52
Incidence of treatment emergent adverse events (TEAE) and serious TEAE
10 years
Changes over time in height Z-score in relation to ACH and non-ACH growth charts
10 years
Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation
Up to 18 months
Change from baseline in annualized height velocity
Up to 18 months
PK parameters of infigratinib (Cmax- PK substudy only)
21 days
PK parameters of infigratinib (Clast- PK substudy only)
21 days
PK parameters of infigratinib (Tmax- PK substudy only)
21 days
PK parameters of infigratinib (AUC24- PK substudy only)
21 days
PK parameters of infigratinib (T1/2- PK substudy only)
21 days
PK parameters of infigratinib (AUCinf- PK substudy only)
21 days
PK parameters of infigratinib (CL/F- PK substudy only)
21 days
PK parameters of infigratinib (Vz/F- PK substudy only)
21 days
PK parameters of infigratinib (Racc- PK substudy only)
21 days

Secondary Endpoints

Change from BL in height Z-score (in relation to ACH tables)
Week 52
Change from BL in upper to lower body segment ratio
Week 52
Change from BL in height Z-score (in relation to non-ACH tables)
Week 52
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Infigratinib 0.25 mg/kg/dayEXPERIMENTALInfigratinib at 2, 3.5, 5, 7, 10 mg
Placebo 0.25 mg/kg/dayPLACEBO_COMPARATORPlacebo Comparator at 2, 3.5, 5, 7, 10 mg
Arm 1: Rollover subjectsEXPERIMENTALChildren who have completed QED-sponsored interventional study with infigratinib (Phase 2 or Phase 3)
Arm 2: Treatment naïve subjectsEXPERIMENTALChildren naïve to infigratinib
Infigratinib 0.016 mg/kgEXPERIMENTALDose Escalation: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Infigratinib 0.032 mg/kgEXPERIMENTALDose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Infigratinib 0.064 mg/kgEXPERIMENTALDose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Infigratinib 0.128 mg/kgEXPERIMENTALDose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Infigratinib 0.25 mg/kgEXPERIMENTALDose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months. Dose Expansion: Upon identification of the recommended dose from all cohorts analyzed, an expansion cohort of 20 subjects may begin enrollment to further determine safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of the selected dose.

Interventions

NameTypeDescription
Infigratinib 0.25 mg/kg/dayDRUGDaily doses of oral Infigratinib (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg
Placebo Comparator 0.25 mg/kg/dayDRUGDaily doses of oral Placebo Comparator (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg
InfigratinibDRUGInfigratinib minitablets or sprinkle capsules to be administered by mouth. In subjects that completed a prior study with infigratinib, the starting dose will be the same as the last dose received in the prior interventional study with infigratinib. Infigratinib dose may be adjusted to 0.25 mg/kg/day (the dose selected to be explored further in the dose escalation portion of Phase 2 study QBGJ398-201 (PROPEL 2)).
Infigratinib 0.016 mg/kgDRUGInitial cohort dose of infigratinib at the protocol-specified starting dose, with subsequent cohort escalations based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
Infigratinib 0.032 mg/kgDRUGSubsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
Infigratinib 0.064 mg/kgDRUGSubsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
Infigratinib 0.128 mg/kgDRUGSubsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
Infigratinib 0.25 mg/kgDRUGSubsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
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Eligibility Criteria

Age Range3 Years to 17 Years
SexALL
Healthy VolunteersNo
Study Sites27

Inclusion Criteria: 1. Subject must be 3 to \<18 years of age at screening with growth potential defined as annualized height velocity of \>1.5 cm/year over a period of at least 6 months of participation in the PROPEL observational study (QBGJ398-001), pubertal Tanner stage ≤4, and bone age ≤13 yea...

Countries:United StatesArgentinaAustraliaCanadaFranceItalyNorwaySingaporeSpainUnited Kingdom
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Frequently asked questions about Infigratinib/kg

What is Infigratinib used for?

Infigratinib is an investigational small molecule being developed for the rare skeletal disorders achondroplasia and hypochondroplasia. It is studied in children and adolescents, with clinical trials enrolling participants as young as 3 years old. The drug is not approved and remains in clinical development.

What does Infigratinib target?

Infigratinib is a kinase inhibitor, belonging to the -tinib class of drugs that target tyrosine kinases. It is being studied for its effects on bone growth in achondroplasia and hypochondroplasia. The specific molecular target is not disclosed in the available information.

Who makes Infigratinib?

Infigratinib is being developed by BridgeBio Pharma, Inc., a biopharmaceutical company traded on NASDAQ under the ticker BBIO. BridgeBio is conducting clinical trials of the drug in multiple countries for achondroplasia and hypochondroplasia.

What phase is Infigratinib in?

Infigratinib is in Phase 2 and Phase 3 clinical trials. It has received FDA designations including Breakthrough Therapy, Orphan Drug, Fast Track, and Rare Pediatric Disease. The drug is investigational and has not been approved by the FDA.

What clinical trials is Infigratinib in?

Infigratinib has been studied in several trials, including NCT04265651, a completed Phase 2 study in achondroplasia with 84 participants, and NCT06164951, a completed Phase 3 study with 114 participants. An extension study, NCT05145010, is enrolling by invitation with a target of 300 participants.

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

Yes, infigratinib 0.128 mg/kg/day and infigratinib/kg/day refer to the same drug, Infigratinib, at specific dosing levels used in clinical studies. These alternative names describe the dose administered per kilogram of body weight per day in pediatric trials.