The Role of the Vosoritide Drug in Childrens with Achondroplasia
Keywords:
Achondroplasia;Vosoritide;Metabolism;Dosage.
Abstract
Achondroplasia is a hereditary disorder that follows an autosomal dominant inheritance pattern. The condition is a result of harmful mutations in the FGFR3 gene, which occur when there are two different versions of the gene presentMutations in the de novo gene FGFR3 prevent the development of chondrocytes forward unregulated growth and instead promote the growth of existing bone,this negatively affects the creation of new bone. Vosoritide, additionally called voxzogo, represents a significant leap approaching the approval of a pharmaceutical treatment that is specifically intended to address achondroplasia. Aforementioned medication has been approved for use in-home injections, these injections must be conducted under the supervision of a knowledgeable nurse in ordering to ensure proper guidance and safety.
References
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Chan ML, Qi Y, Larimore K, et al. Pharmacokinetics and exposureresponse
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Hoover-Fong J, Cheung MS, Fano V, et al. Lifetime impact of achondroplasia: current evidence and perspectives on the natural history. Bone. 2021;146: 115872.
Lorne H, Newman CJ, Unger S. Is height important for quality of life in children with skeletal dysplasias? Eur J Med Genet. 2020;63(4): 103816.
McGraw SA, Henne JC, Nutter J, Larkin AA, Chen E. Treatment goals for achondroplasia: a qualitative study with parents and adults. AdvTher. 2022;39(7):3378–91.
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Pfeiffer KM, Brod M, Smith A, et al. Assessing physical symptoms, daily functioning, and wellbeing in children with achondroplasia. Am J Med Genet A. 2021;185(1):33–45.
Pfeiffer KM, Brod M, Smith A, Viuff D, Ota S, Charlton RW. Functioning and well-being in older children and adolescents with achondroplasia: a qualitative study. Am J Med Genet A. 2022;188(2): 454–62.
Qi, Y. (2024). Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model. Clinical Pharmacokinetics, 63(5), 707–719. https://doi.org/10.1007/s40262-024-01371-6
Savarirayan R, Tofts L, Irving M, et al. Safe and persistent growthpromoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study. Genet Med. 2021;23(12):2443-2447.
Saitou, H. (2024). Clinical outcomes and medical management of achondroplasia in Japanese children: A retrospective medical record review of clinical data. American Journal of Medical Genetics, Part A, 194(8). https://doi.org/10.1002/ajmg.a.63612
Savarirayan, R. (2021). Vosoritide treatment accelerates bone growth in children with achondroplasia. Future Rare Diseases, 1(3). https://doi.org/10.2217/frd-2021-0009
Savarirayan, R. (2024). New treatments for children with achondroplasia. The Lancet Child and Adolescent Health, 8(4), 301–310. https://doi.org/10.1016/S2352-4642(23)00310-3
Shediac R, Moshkovich O, Gerould H, et al. Experiences of children and adolescents living with achondroplasia and their caregivers. Mol Genet Genomic Med. 2022;10(4):e1891.
Taylor-Miller, T. (2024). Progress in managing children with achondroplasia. Expert Review of Endocrinology and Metabolism. https://doi.org/10.1080/17446651.2024.2390416
Tofts, L. (2024). Consensus Guidelines for the Use of Vosoritide in Children with Achondroplasia in Australia. Children, 11(7). https://doi.org/10.3390/children11070789
U.S. Food and Drug Administration. Highlights of prescribing information. VOXZOGO (vosoritide) for injection, for subcutaneous use. Accessed August 8, 2022.
Zikeli, S. (2022). Achondroplasia in children: Vosoritide as the first specific therapeutic agent. Medizinische Monatsschrift Fur Pharmazeuten, 45(5), 193–194. https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85133542850&origin=inward
Chan ML, Qi Y, Larimore K, et al. Pharmacokinetics and exposureresponse
of vosoritide in children with achondroplasia. ClinPharmacokinet. 2022;61(2):263-280.
European Medicines Agency. Voxzogo 2022 [updated 2022 February]. https://www.ema.europa.eu/en/medicines/human/EPAR/voxzogo. Accessed 13 Mar 2023.
Ireland P. O’Donaghey S. Development of Australian children with achondroplasia. Journal of Developmental and BehaviouralPaediatrics. 2007; 31(1): 41-7.
Hoover-Fong J, Cheung MS, Fano V, et al. Lifetime impact of achondroplasia: current evidence and perspectives on the natural history. Bone. 2021;146: 115872.
Lorne H, Newman CJ, Unger S. Is height important for quality of life in children with skeletal dysplasias? Eur J Med Genet. 2020;63(4): 103816.
McGraw SA, Henne JC, Nutter J, Larkin AA, Chen E. Treatment goals for achondroplasia: a qualitative study with parents and adults. AdvTher. 2022;39(7):3378–91.
Pauli RM. Achondroplasia: a comprehensive clinical review. Orphanet J Rare Dis. 2019;14(1):1.
Pfeiffer KM, Brod M, Smith A, et al. Assessing physical symptoms, daily functioning, and wellbeing in children with achondroplasia. Am J Med Genet A. 2021;185(1):33–45.
Pfeiffer KM, Brod M, Smith A, Viuff D, Ota S, Charlton RW. Functioning and well-being in older children and adolescents with achondroplasia: a qualitative study. Am J Med Genet A. 2022;188(2): 454–62.
Qi, Y. (2024). Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model. Clinical Pharmacokinetics, 63(5), 707–719. https://doi.org/10.1007/s40262-024-01371-6
Savarirayan R, Tofts L, Irving M, et al. Safe and persistent growthpromoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study. Genet Med. 2021;23(12):2443-2447.
Saitou, H. (2024). Clinical outcomes and medical management of achondroplasia in Japanese children: A retrospective medical record review of clinical data. American Journal of Medical Genetics, Part A, 194(8). https://doi.org/10.1002/ajmg.a.63612
Savarirayan, R. (2021). Vosoritide treatment accelerates bone growth in children with achondroplasia. Future Rare Diseases, 1(3). https://doi.org/10.2217/frd-2021-0009
Savarirayan, R. (2024). New treatments for children with achondroplasia. The Lancet Child and Adolescent Health, 8(4), 301–310. https://doi.org/10.1016/S2352-4642(23)00310-3
Shediac R, Moshkovich O, Gerould H, et al. Experiences of children and adolescents living with achondroplasia and their caregivers. Mol Genet Genomic Med. 2022;10(4):e1891.
Taylor-Miller, T. (2024). Progress in managing children with achondroplasia. Expert Review of Endocrinology and Metabolism. https://doi.org/10.1080/17446651.2024.2390416
Tofts, L. (2024). Consensus Guidelines for the Use of Vosoritide in Children with Achondroplasia in Australia. Children, 11(7). https://doi.org/10.3390/children11070789
U.S. Food and Drug Administration. Highlights of prescribing information. VOXZOGO (vosoritide) for injection, for subcutaneous use. Accessed August 8, 2022.
Zikeli, S. (2022). Achondroplasia in children: Vosoritide as the first specific therapeutic agent. Medizinische Monatsschrift Fur Pharmazeuten, 45(5), 193–194. https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85133542850&origin=inward
Published
2024-09-19
How to Cite
Ahmed, A. T., Hameed, I. H., & Hameed, N. H. (2024). The Role of the Vosoritide Drug in Childrens with Achondroplasia. Central Asian Journal of Medical and Natural Science, 5(4), 835-840. Retrieved from https://cajmns.centralasianstudies.org/index.php/CAJMNS/article/view/2615
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Articles
