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The FGFR3 dwarfism gene can be turned off with injections

The FGFR3 dwarfism gene can be turned off with injections

The protein encoded by the FGFR3 gene inhibits the division of growth plate cells.

Normally, it works to prevent very high growth, but with mutations, a person grows very short.

In 2021, BioMarin created the drug called Vosoritide, which regulates FGFR3. It contains a C-type natriuretic peptide (CNP) analog. This substance turns off the creation of the FGFR3 protein.

The drug was approved by the EMA and FDA for the treatment of dwarfism and is already in use.

Treatment of children with achondroplasia with this drug

… resulting in a statistically significant improvement in annualized growth velocity of 1.57 cm/year.1

As a result, scientists predict that children will reach the same heights as their healthy peers.

Will it work on healthy people?

If the growth zones are still unfused, probably yes.

CNP acts as a growth stimulant, not only in sick people. Overexpression of CNP in humans and animals is associated with enhanced skeletal growth, while insufficient expression leads to growth delay.2

But since the drug is new, its impact on healthy people is not yet well studied.

Consult with specialists for treatment possibilities.

  1. BioMarin Receives FDA Approval for VOXZOGO™ (vosoritide) for Injection, Indicated to Increase Linear Growth in Children with Achondroplasia Aged 5 and Up with Open Growth Plates ↩

  2. Allen, D. B., Merchant, N., Miller, B. S., & Backeljauw, P. F. (2021). Evolution and Future of Growth Plate Therapeutics. Hormone research in paediatrics, 94(9-10), 319–332. https://doi.org/10.1159/000520812 ↩

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