英菲格拉替尼治疗软骨发育不全吗？

Treating Achondroplasia

Achondroplasia is the most common short-limb skeletal dysplasia caused by gain-of-function pathogenic variants in the fibroblast growth factor receptor 3 (FGFR3) gene, a negative regulator of endochondral bone formation. Most treatment options are symptomatic and target medical complications.

Infigratinib, an orally bioavailable FGFR1-3 selective tyrosine kinase inhibitor, is being studied as a direct therapeutic strategy to combat FGFR3 hyperactivity in patients with achondroplasia. Infigratinib has certain safety and efficacy in the treatment of achondroplasia.

Infigratinib other drug indications

In addition to treating achondroplasia, infigratinib is indicated for the treatment of unresectable locally advanced or metastatic intrahepatic cholangiocarcinoma.

Infigratinib is a fibroblast growth factor receptor (FGFR)-specific tyrosine kinase inhibitor that can inhibit FGFR activity, reduce cancer cell proliferation and cause tumor cell death, thereby exerting its effect.

The FDA has approved the FGFR2 inhibitor infigratinib for patients who have received treatment for locally advanced or metastatic cholangiocarcinoma. In a phase II study, the drug had an overall response rate of 23% and a progression-free survival of 7.3 months. About 77% of patients developed hyperphosphatemia, the drug's most common side effect and a known target toxicity of FGFR inhibitors.

It should be noted that patients should use infigratinib under the evaluation of a doctor and should not use the drug privately to avoid adverse drug reactions, such as hyperphosphatemia, dry eye syndrome, nail toxicity, stomatitis, fatigue, hair loss, diarrhea, abdominal pain, dry skin, loss of appetite, joint pain, constipation, blurred vision and vomiting, etc., which may affect physical health.

If patients experience serious adverse reactions during treatment, they should consult a doctor in time and deal with it, such as discontinuing the drug, adjusting the dose, or switching to other drug treatments.

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