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First drug for Childhood achondroplasia is expected to come soon

First drug for Childhood achondroplasia is expected to come soon, bringing new life to “dwarfism”.

Chondrodysis is the most common and disproportionate type of short stature in humans, and is also called “dwarfism.”

Recently, at the 2021 American College of Medical Genetics and Genomics (ACMG) annual clinical genetics conference, the C-type natriuretic peptide analogue Vosoritide performed brilliantly:

In a phase 2 clinical trial, after 5 years of treatment (60 months), the growth rate of the children continued to improve, and the total duration of the growth phase did not shorten. Moreover, the patient’s bone age progressed normally, and the annual X-ray showed no significant changes in bone mineral content and bone density.

Last year, Vosoritide submitted a New Drug Application (NDA) to the US FDA, and it is expected to receive an approval response on August 20 this year. This means that in four months, children with achondroplasia are expected to usher in the first treatment drug.

▌Chondroplasia

Chondrodysplasia is the most common and disproportionate type of short stature in humans, with an average of 1 in 25,000 babies. It is characterized by the slowing of intrachondral ossification, leading to disproportionate short stature and structural disorders of the skull, spine, face and skull base, which is a kind of dwarfism. The representative of such patients is probably the “little devil” in “Game of Thrones”.

First drug for Childhood achondroplasia is expected to come soon

Among children with achondroplasia, more than 80% of the parents are of normal height; but as long as one of the parents has the disease-causing gene, the child has a 1/2 chance of getting the disease.

In addition to short stature, people with achondroplasia can also experience serious health complications, including sleep apnea, bent legs, facial hypoplasia, spinal stenosis, and recurrent ear infections.

For a long time, patients with achondroplasia have lacked an effective treatment plan, only using growth hormone to treat, the effect of growing taller is not ideal. At present, there are some invasive surgical treatments to lengthen bones and cope with some complications, such as limb lengthening and correction, spinal cord decompression, and arc leg straightening surgery, but the risks and costs of surgery are greater.

Studies have shown that the mortality rate of these children in each age group is increasing. At present, there is no drug approved for the treatment of achondroplasia in the world, and patients have long faced the dilemma of “no medicine to cure”.

▌C-type natriuretic peptide analogue: Vosoritide

Vosoritide is a C-type natriuretic peptide (CNP) analog derived from natural human peptide. Natural human peptide is a positive regulator of bone growth and can stimulate endochondral ossification.

As a genetic disease, achondroplasia is caused by mutations in the FGFR3 gene. FGFR3 is a negative regulator of bone growth. Mutations in this gene can inhibit the proliferation and differentiation of chondrocytes in the patient’s growth plate, leading to long bone growth disorders.

By inhibiting the overactive FGFR3 pathway, CNP can stimulate the proliferation and differentiation of growth plate chondrocytes, thereby promoting the growth of long bones caused by cartilage internalization.

Prior to this, Vosoritide has also been awarded the title of “Orphan Drug” and priority review by the FDA for the treatment of childhood achondroplasia.

Currently, the drug is being evaluated in children (18 years and older) whose growth plates are still in an “open” state. These patients account for approximately 25% of achondroplasia.

▌ “Raise up” every year, children with short stature become taller!

The latest data from an ongoing, open-label Phase 2 clinical extension trial shows:

During the 60-month treatment period, the average increase in annual growth rate (AGV) (±SD) was 1.35 (±1.07) cm/year.

Using the CDC standard of children’s average height, at 60 months, the total average (±SD) of children’s height Z score increased by 0.78 (±0.70).

Moreover, at all doses, Vosoritide is well tolerated and safe.

Previously, the results of a one-year phase 3 clinical trial showed that the average annual length of children treated with Vosoritide was 1.57 cm, and the growth rate basically caught up with their peers.

Both trials have obtained positive results. Children with achondroplasia are “uplifted” every year, and the annual growth rate not only catches up with their peers, but also continues to improve.

Once approved, 　　 Vosoritide will become the first drug to treat achondroplasia, bringing new life to patients with “dwarfism”

(source:internet, reference only)

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