FDA approves first therapy for dwarfism

USA – BioMarin Pharmaceutical Inc’s once-daily injection for children with the most common type of dwarfism has been approved by the US Food and Drug Administration, making it the country’s first approved therapy for achondroplasia.

BioMarin Pharmaceutical developed the treatment, which is a once-daily injection for children with achondroplasia, a rare genetic disorder that causes dwarfism and can lead to serious medical complications.

Patients who received the drug, called Voxzogo, grew 1.6 centimeters more over the course of a year than those who received a placebo in a pivotal clinical trial.

According to BioMarin, patients who take Voxzogo throughout childhood are more likely to reach heights comparable to their peers who do not have achondroplasia.

The approval of Voxzogo, which is intended for children aged five and up, comes months after it became the first therapy approved by European regulators for the treatment of the genetic condition.

Many countries still lack an approved treatment for Achondroplasia, a condition characterized by short stature that affects approximately one in every 25,000 newborns worldwide.

To thousands of small people, achondroplasia’s short stature is not a disability that must be treated, but rather a unique feature to be celebrated.

Voxzogo is approved for children aged 5 to 18 with achondroplasia. BioMarin is currently enrolling infants and young children in a study, with results expected next year. If the results are favorable, the company intends to apply for a broader FDA label.

The drug developed by BioMarin is intended to counteract the genetic mutation that causes achondroplasia. The condition is caused by a mutation in the fibroblast growth factor receptor (FGFR3) gene, which controls bone growth.

Achondroplasia disrupts the natural process by which cartilage turns into bone, resulting in shorter stature, a bowing of the spine, and the risk of serious complications that necessitate surgery.

BioMarin anticipates that the therapy will generate approximately US$240,000 in annual net revenue per patient in the United States, and that approximately 3,000 children with achondroplasia in the country will be eligible for the treatment.

The company expects Voxzogo to be available in the United States by mid-to-late December at a list price of US$899 per vial.

BioMarin’s late stage study of Voxzogo demonstrated improvements in patients’ growth rates over a year and was well tolerated.

The drug is also being studied in children under the age of five, and it is already approved in Europe for those aged two and up.

The Food and Drug Administration of the United States extended the drug’s review by three months in April to assess two-year data on the drug.

It has now cleared the drug through the accelerated approval pathway, which necessitates a post-marketing study to determine final adult height.

BioMarin stated that it intended to receive final approval based on the results of ongoing extension studies.

According to BioMarin, the drug is also being reviewed by regulatory authorities in Japan, Brazil, and Australia, and could be approved in those countries next year.

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