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Fgfr3

FGFR3 is a fibroblast growth factor receptor whose constitutively active mutation causes achondroplasia (most common dwarfism).

Aliases (5)
Voxzogo · vosoritide (BMN 111) · infigratinib (Truseltiq, BBP-831) · sotuletinib (TAS-120 / futibatinib variant) · BGJ398
TYPICAL DOSE
15 µg/kg SC once daily, while growth plates are…
Daily
ROUTE
CYCLE
STORAGE

Overview

What is Fgfr3?

FGFR3 (Fibroblast Growth Factor Receptor 3) is a receptor tyrosine kinase involved in bone growth, hair follicle cycling, and cellular signaling. As a research target, FGFR3-modulating compounds and peptides are explored for hair loss, cartilage disorders, and certain cancers.

Key Benefits

FGFR3 modulators are investigated for treatment of achondroplasia (loss-of-function approaches), hair regrowth (gain-of-function in follicle), and oncology (FGFR3-driven tumors). Not yet a consumer compound.

Mechanism of Action

FGFR3 is a transmembrane receptor tyrosine kinase activated by fibroblast growth factors. Downstream signaling via RAS-MAPK and PI3K-AKT regulates chondrocyte proliferation, hair follicle stem cells, and cell survival.

Pharmacokinetics

·
PeakHalf-life
Approximate curve — visual aid only, not data-precise PK

What to Expect

  • Week 1
    Tolerability and dose-response.
  • Week 2-4
    Early effect window.
  • Week 4-8
    Peak benefit assessment.
  • Week 8+
    Cycle decision point.

Side Effects & Safety

  • Common (>10%): Vosoritide — injection site reaction, transient mild hypotension, dizziness. TKIs — hyperphosphatemia, GI upset, retinal pigment epithelial detachment.
  • Less common (1-10%): Vomiting, fatigue (vosoritide). TKIs — hand-foot syndrome, nail toxicity, transaminitis.
  • Rare-serious (<1%): TKIs in oncology dose — serous retinal detachment, severe hyperphosphatemia, cardiotoxicity.
  • Specific watch periods: Vosoritide — first 30 min post-injection for symptomatic hypotension; long-term — joint growth proportions monitored.

References

Savarirayan R, et al. (2020) — Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial. Lancet

pubmed.ncbi.nlm.nih.gov · 2020

PMID 32945803, vosoritide pivotal trial

View Study

Savarirayan R, et al. (2022) — Vosoritide for children with achondroplasia: a 30-month update from an ongoing extension study. Genetics in Medicine

pubmed.ncbi.nlm.nih.gov · 2022

PMID 35305862, long-term extension

View Study

Komla-Ebri D, et al. (2016) — Tyrosine kinase inhibitor NVP-BGJ398 functionally improves FGFR3-related dwarfism in mouse model. J Clin Invest

pubmed.ncbi.nlm.nih.gov · 2016

PMID 27348590, infigratinib mechanism in achondroplasia model

View Study

Goyal L, et al. (2023) — Futibatinib for FGFR2-rearranged intrahepatic cholangiocarcinoma. NEJM

pubmed.ncbi.nlm.nih.gov · 2023

PMID 36534649, futibatinib (sotuletinib alias) oncology pivotal — confirms it is NOT an achondroplasia drug

View Study

Foreman PK, et al. (2020) — Birth prevalence of achondroplasia: a systematic literature review. Am J Med Genet A

pubmed.ncbi.nlm.nih.gov · 2020

PMID 32243066, epidemiologic context

View Study
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