C-type natriuretic peptide analogue vosoritide treatment continued to maintain growth levels for 2 years

BioMarin is a global biotechnology company dedicated to the development and commercialization of innovative therapies for patients suffering from serious and life-threatening rare and ultra-rare genetic diseases.

Vosoritide is a C-type natriuretic peptide (CNP) analog that is injected once a day for the treatment of childhood achondroplasia, which is the most common disproportionate short stature in humans.

Currently, vosoritide is undergoing priority review by the US FDA and review by the EU EAM.

If approved, vosoritide will become the first drug to treat achondroplasia.

The open label expansion data released at the ENDO21 annual meeting showed that children maintained their annual growth rate (AGV) growth during the second year of continuous vosoritide treatment.

In the open-label extension group, children who switched from placebo to vosoritide had similar efficacy to children who received vosoritide one year after treatment.

The retention rate of patients receiving treatment is very high.

Two-year data show that vosoritide is well tolerated at a dose of 15μg/Kg/day, and there are no new safety findings.

Melita Irving, a researcher in the vosoritide clinical project and a clinical geneticist at the Guy's and St Thomas' NHS Foundation Trust in the United Kingdom, said: “Maintaining a sustained level of growth is important because it supports vosoritide in addressing cartilage The root cause of hypoplasia.

The mechanism of action of vosoritide (click on the picture to see a larger picture)

Achondroplasia is the most common disproportionate short stature in humans.

In addition to disproportionately short stature, patients with achondroplasia may experience serious health complications, including foramen compression, sleep apnea, bent legs, facial hypoplasia, permanent lower back swings, spinal stenosis, and repetitive ears.

More than 80% of the parents of children with achondroplasia are of medium stature, and the disease is caused by spontaneous genetic mutations.

Vosoritide is a C-type natriuretic peptide (CNP) analog derived from natural human peptides and is an effective stimulator of endochondral ossification.

Currently, vosoritide is being evaluated for children whose growth plate is still "open", usually children under 18 years of age, and these patients account for approximately 25% of patients with achondroplasia.

Original source: BioMarin Announces Oral Presentation at ENDO2021, the Endocrine Society's Annual Meeting, with Data Demonstrating 2 Years of Treatment Benefit in Children with Achondroplasia Treated with Vosoritide