BioMarin has recently submitted a Marketing Authorization Application to the European Medicines Agency for their achondroplasia treatment, vosoritide. As of now, the review of the application will begin in August. If all goes well, vosoritide will be the first pharmacological treatment for achondroplasia.
Achondroplasia is a disorder of the bones that causes dwarfism. This means that affected individuals are short in stature even as adults. The average height of a female with this condition is 4″1, while the average height of a male is 4″4. Along with the physical symptoms, people with achondroplasia experience decreased muscle tone, hydrocephalus, spinal stenosis, and apnea. All of these symptoms are the result of a mutated FGFR3 gene, which is either inherited in an autosomal dominant pattern or sporadic. This mutation causes the over activity of a certain protein, leading to abnormal skeletal development.
Vosoritide for Achondroplasia
Vosoritide is an injection analog of C-type Natriuretic Peptide that is intended to be administered once a day. It is a treatment for children with achondroplasia. After a placebo-controlled, double-blind, randomized Phase 3 trial, BioMarin submitted their MAA application to the European Medicines Agency. This study found that vosoritide maintained its safety and efficacy profiles.
In addition to this application, the biotechnology company plans to submit a New Drug Application to the FDA in the third quarter of this year.
If both of these applications are successful, there will finally be a pharmacological treatment option for those with achondroplasia. It will address the underlying cause of the condition and hopefully reduce the complications associated with it. If approved, it will better the lives of those with achondroplasia.
Read more about vosoritide here.