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Update and review of treatment in achondroplasia
Silvia Gallego-Gutiérrez1, Ana Coral Barreda-Bonis2, Isolina Riaño-Galán3
1Unidad de Endocrinología Pediátrica, Hospital Regional de Málaga, Málaga, Spain; Programa de Doctorado en Biomedicina, Investigación Translacional y Nuevas Tecnologías en Salud, Facultad de Medicina, Universidad de Málaga, Málaga, Spain; Instituto de Investigación Biomédica de Málaga (IBIMA) - Plataforma Bionand, Málaga, Spain.
Insights
Achondroplasia treatments are evolving with the introduction of targeted therapies like vosoritide. This shift impacts growth, final height, and associated health issues, necessitating updated management strategies.
Area of Science:
- Genetics and skeletal biology
- Pediatric endocrinology
- Pharmacology
Background:
- Achondroplasia, the most common skeletal dysplasia, results from FGFR3 gene variants disrupting ossification.
- It affects approximately 4.6 per 100,000 births, causing disproportionate short stature.
- Traditional management has been largely supportive.
Purpose of the Study:
- To offer an updated overview of achondroplasia therapeutic approaches.
- To cover currently approved treatments and those under investigation.
- To highlight the impact of new therapies on growth and comorbidities.
Main Methods:
- Review of current literature and clinical trial data.
- Analysis of outcomes from patients treated with vosoritide.
- Examination of emerging therapeutic strategies.
Main Results:
- Vosoritide's 2021 approval marks a significant change in achondroplasia management.
- Initial outcomes show impacts on growth, final height, and comorbidities like foramen magnum stenosis.
- Other novel therapeutic strategies are under development.
Conclusions:
- Targeted therapies are transforming achondroplasia care.
- Continuous updates on approved and investigational treatments are crucial.
- The focus is expanding beyond height to overall health and body proportions.
Introduction:
Achondroplasia is the most common skeletal dysplasia associated with disproportionate short stature, with an estimated prevalence of 4.6 per 100 000 births. It is caused by a pathogenic variant in the gene encoding fibroblast growth factor receptor 3 (FGFR3), which disrupts endochondral ossification of the growing skeleton.
Objective:
To provide an updated overview of the therapeutic approach to achondroplasia, including currently approved treatments and those under investigation.
Content:
The approval of vosoritide in 2021 has brought a major shift in the management of these patients. Updated follow-up guidelines have been published, along with initial outcomes in treated patients, with the aim of determining not only the impact on growth and final height but also on associated comorbidities, such as foramen magnum stenosis, and body proportions. We also review other emerging therapeutic strategies currently under development.
Conclusions:
The availability of targeted therapies has modified the traditional approach to the management of achondroplasia, which makes ongoing updates on approved and investigational treatments essential.
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