Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Infigratinib
Infigratinib /kg · 3 trials · 1 indication
| Arm | Type | Description |
|---|---|---|
| Infigratinib 0.25 mg/kg/day | EXPERIMENTAL | Infigratinib at 2, 3.5, 5, 7, 10 mg |
| Placebo 0.25 mg/kg/day | PLACEBO_COMPARATOR | Placebo Comparator at 2, 3.5, 5, 7, 10 mg |
| Arm 1: Rollover subjects | EXPERIMENTAL | Children who have completed QED-sponsored interventional study with infigratinib (Phase 2 or Phase 3) |
| Arm 2: Treatment naïve subjects | EXPERIMENTAL | Children naïve to infigratinib |
| Infigratinib 0.016 mg/kg | EXPERIMENTAL | Dose 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/kg | EXPERIMENTAL | Dose 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/kg | EXPERIMENTAL | Dose 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/kg | EXPERIMENTAL | Dose 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/kg | EXPERIMENTAL | Dose 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. |
| Name | Type | Description |
|---|---|---|
| Infigratinib 0.25 mg/kg/day | DRUG | Daily doses of oral Infigratinib (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg |
| Placebo Comparator 0.25 mg/kg/day | DRUG | Daily doses of oral Placebo Comparator (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg |
| Infigratinib | DRUG | Infigratinib 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/kg | DRUG | Initial 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/kg | DRUG | Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth. |
| Infigratinib 0.064 mg/kg | DRUG | Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth. |
| Infigratinib 0.128 mg/kg | DRUG | Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth. |
| Infigratinib 0.25 mg/kg | DRUG | Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth. |
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...
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.
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.
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.
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.
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.
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.