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Vosoritide: treating achondroplasia, and the state of the research

Clinical review pending

Vosoritide is meant to boost growth in height in children with achondroplasia. How it works and what risks come with treatment.

What is vosoritide?

Vosoritide, brand name Voxzogo, is a modified analog of C-type natriuretic peptide (CNP), a messenger molecule involved in bone growth. It is used to boost growth in height in children with achondroplasia. In this genetic condition, an overactive FGFR3 signaling pathway holds back bone growth at the growth plates. Vosoritide was developed under the code BMN-111 and approved in the US in 2021.

How does vosoritide work?

In achondroplasia, a mutation keeps the FGFR3 receptor permanently switched on. The signals it sends downstream hold back the growth of cartilage cells at the growth plate and so limit how much the bones lengthen. Vosoritide binds to a different receptor, natriuretic peptide receptor B. This raises cyclic GMP in the cartilage cells, which inhibits the overactive FGFR3 pathway. In this way, the brake on growth can be partly released and growth of the long bones promoted.

Natural CNP is broken down within a few minutes. Vosoritide was modified so that it can be given daily. In the phase 3 trial, children on vosoritide grew about 1.6 cm more per year than children on placebo 1. The treatment is used while the growth plates are still open. One known effect is a temporary drop in blood pressure, which is why children are given food and fluids before each dose. Source

Is vosoritide FDA-approved?

Yes. The FDA approved vosoritide as Voxzogo in 2021 for achondroplasia in children with open epiphyses, meaning open growth plates 2. This accelerated approval is based on a higher annual growth velocity. Whether it continues may depend on confirmatory trials. The approval originally applied from age 5. Since October 2023, it has covered children of all ages with open growth plates.

What is vosoritide prescribed for?

Vosoritide is meant to increase growth in height in children with achondroplasia whose growth plates are still open. Once the growth plates have closed, treatment is stopped, because the drug depends on active bone growth.

What side effects have been reported?

According to the prescribing information, common side effects include:

  • Injection-site reactions.

  • Vomiting.

  • A temporary drop in blood pressure.

  • Joint pain. Source

In children under 5, rash is also common.

The more serious risks include:

  • Low blood pressure. To limit this risk, care is taken before each dose to make sure the child has eaten and had something to drink.

References provided in the source article

Sources: FDA prescribing information for Voxzogo; the phase 3 vosoritide trial (Savarirayan et al., The Lancet, 2020, DOI: 10.1016/S0140-6736(20)31541-5 1 ), indexed on PubMed.

Provenance and editorial review status

Adapted for Nutrient Desk from the source article. The claims and source attributions in the original text still require clinical review. This draft has not been cleared for indexing.