Abbisko Therapeutics reports positive Phase II results for achondroplasia drug

Abbisko Therapeutics Co., Ltd. has reported preliminary Phase II study results for lavengratinib (ABSK061), an oral FGFR2/3 inhibitor aimed at treating achondroplasia in children aged 3 to 12.

Data from the first and lowest-dose cohort, involving seven children aged 6 to 12, showed a mean increase of 2.4 centimeters per year in annualized height velocity after 27 weeks of treatment. All participants in this cohort met the study's responder criteria.

The investigational therapy has demonstrated a favorable safety profile thus far, with no serious adverse events or treatment discontinuations reported. Researchers noted an absence of common safety issues associated with FGFR pathway inhibition, such as corneal toxicity or hyperphosphatemia.

Lavengratinib is being administered via a proprietary oral mini-tablet formulation designed for pediatric use. The company expects to release six-month efficacy and safety results from higher-dose cohorts by the end of 2026.