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Iron
- Joined
- May 11, 2025
- Posts
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infigratinib is a cancer medication that inhibits FGFR1‑FGFR4 signaling.
Infigratinib was studied in the Phase 2 PROPEL 2 trial (NCT04265651), involving 72 children aged 3–11 with achondroplasia. The key findings:
The downside?
Infigratinib was studied in the Phase 2 PROPEL 2 trial (NCT04265651), involving 72 children aged 3–11 with achondroplasia. The key findings:
- Dosages: daily oral doses escalating across cohorts: 0.016, 0.032, 0.064, 0.128, and 0.25 mg/kg. The 0.25 mg/kg/day dose (Cohort 5) was selected for extended treatment reddit.com+10nejm.org+102minutemedicine.com+10.
- Efficacy: In Cohort 5, the mean increase in annualized height velocity reached +3.03 to +3.38 cm/year at 6 months, stabilizing at +2.50 cm/year by month 18; height z‑scores improved by +0.54 and body proportions also enhanced investors.com+9gimopen.org+9globenewswire.com+9.
- Side Effects: All participants experienced ≥1 treatment-emergent adverse event (TEAE), mostly mild-to-moderate. The most common included:
- Common Cold (~40% of children) gimopen.org+62minutemedicine.com+6reddit.com+6
- Other frequent issues: general infections (often upper respiratory), headaches, gastrointestinal discomfort (dyspepsia, flatulence), reduced appetite, hypercholesterolemia, hyperphosphatemia, and decreased vitamin D levels. No serious adverse events, no dose reductions or discontinuations due to AEs investor.bridgebio.com+8pmc.ncbi.nlm.nih.gov+8investor.bridgebio.com+8.
The downside?
- Infection (who cares.)
- Diarrhea, constipation
- Appetite loss
- Headache
- Theoretical Alopecia
- Theoretical Dry eye, blurred vision, REVERSIBLE RETINAL DETACHMENT
- Theoretical 32% chance of serious adverse effects
- Theoretical permanent blindness