Biotech Industry Liaison Committee Review

Medical management of achondroplasia:

Medical management is focused on preventing, anticipating, identifying, and correcting complications as they arise and encouraging a healthy lifestyle, positive self-esteem, and mental health.2 The complications associated with achondroplasia can vary greatly in comparison to others with achondroplasia and across one’s lifetime.2,5

Some individuals experience very minor complications and require little to no medical intervention, while others will require more intensive management, including interventions such as spinal decompression, leg straightening, spinal fusion, and ear tubes. Additionally, the type of complications individuals with achondroplasia may face can change throughout one’s life. As a baby, the major complications include constriction of the nerves in the neck (i.e., foramen magnum) and breathing restrictions. Ear infections, obesity, obstructive apnea, orthopedic concerns, and other complications may also arise throughout childhood & adulthood.2

Until recently, there have been no pharmaceutical therapies approved for achondroplasia. Human growth hormone has been used previously but is not recommended as it does not reduce any of the complications associated with achondroplasia. Furthermore, the small 1 to 1.5-inch increase in height requires years of injections.2,4 Development of targeted therapy designed specifically for achondroplasia is ongoing. Vosoritide is the first FDA approved for achondroplasia and was approved under accelerated approval for use in the United States in November 2021. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory studies. This paper summarizes the research process and the therapies in development.4