Therachon has dosed the first patient in a Phase l trial of TA-46 for treatment of achondroplasia, the most common form of short-limb dwarfism.
The randomised, placebo-controlled, double-blind trial expects to enrol around 70 healthy male and female volunteers.
The study is designed to examine the safety, tolerability and pharmacokinetics of single and multiple ascending doses of TA-46, a soluble recombinant human fibroblast growth factor receptor 3 (FGFR3) ligand trap.
TA-46 is capable of normalising the overactive FGFR3 signalling pathways that underlie bone abnormalities related to achondroplasia.
It is currently under development to serve as a weekly subcutaneous drug for children and adolescents living with achondroplasia.
The Phase I trial will be conducted in the Netherlands.
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Therachon chief medical officer Christian Meyer said: “We are eager to quickly move into the next phase of development, where we aim to demonstrate the unique disease-modifying properties of TA-46 in children with achondroplasia.
“We believe TA-46 has the potential to improve anatomical proportions and thereby prevent the most severe disabling complications of the condition.”
In June last year, Therachon received orphan drug designation from the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) for using TA-46in the treatment of achondroplasia.
Achondroplasia is estimated to affect about one in 15,000 children and is caused by a genetic mutation of the FGFR3, which stunts child bone growth.