ESPE2023 Rapid Free Communications Growth and syndromes (to include Turner syndrome) (6 abstracts)
Real-world safety and effectiveness of vosoritide: Results from an early access program in France
Valérie Cormier-Daire 1 , Thomas Edouard 2 , Bertrand Isidor 3 , Shelda Cohen 4 , Swati Mukherjee 4 , Jeanne Pimenta 4 , Leila Lhaneche 5 , Massimiliano Rossi 6 , Elise Schaefer 7 , Erin Goodman 8 , Sabine Sigaudy 9 & Geneviève Baujat 10
1Centre of Reference for Constitutional Bone Diseases (MOC), Department of Clinical Genetics, Paris Centre University, INSERM UMR 1163, Imagine Institute, Necker-Enfants Malades Hospital, Paris, France. 2Endocrine, Bone Diseases and Genetics Unit, Reference Centre for Rare Diseases of Calcium and Phosphate Metabolism, ERN BOND, OSCAR Network, Paediatric Research Unit, Children’s Hospital, Toulouse University Hospital, RESTORE, INSERM U1301, Toulouse, France. 3Centre Hospitalier Universitaire de Nantes, Nantes, France. 4BioMarin (U.K.) Limited, London, United Kingdom. 5BioMarin (France), Paris, France. 6Service de génétique, Hospices Civils de Lyon; INSERM U1028, CNRS UMR5292, CRNL, GENDEV Team, UCBL1, Lyon, France. 7CHU de Strasbourg - Hôpital de Hautepierre, Strasbourg, France. 8BioMarin Pharmaceutical Inc., Novato, USA. 9Département de Génétique Médicale, Hôpital Timone Enfant, Marseille, France. 10Hopital Necker - Enfants Malades Hopital Necker Enfants Malades, Paris, France
Introduction: Achondroplasia is the most common skeletal dysplasia, in which the main clinical feature is short stature. Vosoritide, the first specific treatment for achondroplasia; administered as a daily subcutaneous injection, was approved by the European Medicines Agency in August 2021 for patients aged ≥2 years until closure of epiphyses. French Health Authorities granted early access to vosoritide treatment in France on 24 June 2021, which continued until commercialization on 13 December 2022. We report the experience of early access to vosoritide during this period led by six referral centers for constitutional bone diseases (MOC) in France.
Methods: In September 2021, early access to vosoritide was initiated in patients aged ≥5 years with open epiphyses. The national center of MOC network (Necker hospital) and five referral centers evaluated patients for treatment. Patients and families received treatment education and were followed-up at month 1, 3, 6 and every 6 months thereafter. Baseline characteristics, data on treatment compliance, safety and efficacy (height, Z-score using CDC reference population) were collected.
Results: A total of 62 patients were enrolled, of which 57 were able to start early access treatment with vosoritide by Dec 2022. The mean age at treatment initiation was 8.6 years (min-max: 5-13 years) and 52% were male. The mean exposure to treatment was 277 days (range 32 - 443 days). 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). After 6 months of treatment (n=37), the mean (SD) annualized growth velocity (AGV) was 5.8 (3.5) cm/year in males (n=21) and 6.3 (2.6) cm/year in females (n=16). 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. Forty-three missed doses were reported by 14 patients and no patients discontinued vosoritide treatment.
Conclusions: These data from 57 children with achondroplasia who were treated for up to 14.5 months (443 days), indicate that vosoritide under real-world conditions has a safety and effectiveness profile consistent with vosoritide clinical trials. Long term data collection for these patients will continue where possible through the EU Voxzogo Post-Authorisation Safety Study (PASS).
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