Achondroplasia clinical trial and regulatory developments

Clinical developments for achondroplasia treatments have shown positive results across different therapeutic approaches.

Abbisko Therapeutics reported preliminary Phase II data for lavengratinib (ABSK061), an oral FGFR2/3 inhibitor for children aged 3 to 12. In the initial low-dose cohort, participants showed a mean increase of 2.4 centimeters per year in annualized height velocity. The therapy demonstrated a favorable safety profile with no serious adverse events or common issues like corneal toxicity reported.

Separately, Phase 3 trial data for navepegritide, a once-weekly TransCon CNP prodrug, indicated improvements in annualized growth velocity, skeletal alignment, and physical function. Long-term follow-up from the ApproaCH trial suggests sustained benefits in lower-extremity alignment, including improved tibial-femoral angles and mechanical axis deviation. While some participants developed antibodies, these did not appear to impact clinical outcomes, and the safety profile remained favorable with low rates of injection-site reactions and no clinically significant hypotension.

Regulatory progress for navepegritide has continued following its February 2026 FDA approval in the United States. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion recommending marketing authorization for TransCon CNP for children aged two and older with achondroplasia whose epiphyses are not closed. A final decision from the European Commission is expected in the fourth quarter of 2026. If authorized, Ascendis Pharma plans to launch the treatment under the brand name YUVIWEL in Germany during the first quarter of 2027.