Description

Real-World Safety and Effectiveness of Vosoritide▼

In August 2021, the EMA approved vosoritide for treating ACH in patients aged ≥2 years until closure of epiphyses. In France, a cohort Temporary Authorisation for Use was granted in June 2021. This cohort transitioned to an authorised early-access program in December 2021.

This poster and oral presentation from ESPE 2023 report the final findings from
this real-world access program. 24 June 2021 to 13 December 2022. Children with molecularly confirmed achondroplasia (N=57) received once-daily, subcutaneous vosoritide 15 µg/kg. Patients were then followed up at Months 1, 3, 6 – and at 6-monthly intervals thereafter. Data was collected on treatment adherence, adverse events, and growth.

A total of 62 patients were enrolled, of which 57 were able to start early access treatment with vosoritide by Dec 2022. Among 22 patients (39%) who completed 12 months of treatment, males (n=10) showed a mean (SD) height increase from baseline of 5.8 (1.7) cm with a Z-score improvement of 1.0 (0.3); females (n=12) showed a mean increase of 6.5 (1.3) cm and a Z-score improvement of 1.2 (0.3). In total, 21 adverse events were reported during the study period. All events were mild and the majority were injection site reactions and vomiting. No serious adverse events were reported. No patients discontinued vosoritide treatment.

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