Innovative therapy to effectively improve the height of children with "dwarfism" approved by the European Union

On August 27, 2021, BioMarin Pharmaceutical announced that the European Commission has approved the listing of Voxzogo (vosoritide)
.

Vosoritide is a C-type natriuretic peptide (CNP) analog that is injected once a day for the treatment of children with achondroplasia from the age of 2 until the growth plate is closed

Achondroplasia is a common type of dwarfism, which is mainly manifested by the slow growth of the bones at the base of the limbs, spine and skull.
Children can only grow about 4 cm per year (normally 6-7 cm)
.

Approximately 250,000 people worldwide are troubled by this disease, but some symptoms can only be resolved through surgery

The pathological mechanism behind the disease is due to mutations in the gene encoding fibroblast growth factor receptor 3 (FGFR3), and FGFR3 protein is overactive
.

FGFR3 is a negative regulator of bone development, which can affect the proliferation, activation and differentiation of chondrocytes

▲In the process of normal bone growth, chondrocytes will continue to form new bones on the growth plate
.

In children with achondroplasia, mutations in the FGFR3 gene interfere with the proliferation and arrangement of chondrocytes, which in turn affects bone growth

The European Union's decision is based on all data from the vosoritide clinical development project, including the results of a one-year randomized double-blind, placebo-controlled phase 3 clinical trial
.

In phase 3 clinical trials, vosoritide can effectively promote bone growth in children with achondroplasia, and it is still effective one year after injection

The Phase 3 trial is further supported by an ongoing Phase 2 dose exploration clinical project with a follow-up period of up to 5 years to prove the long-term safety and efficacy of the drug
.

The data showed that during the 5-year observation period, the subject’s growth rate continued to be higher than the baseline and higher than the expected annual growth rate, and no bone age acceleration was observed, indicating that vosoritide did not shorten the total growth duration

Reference materials:

[1] European Commission Approves BioMarin's Voxzogo® (vosoritide) for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close.