Vosoritide Is One Step Closer to Approval for Achondroplasia in Europe

BioMan Pharmaceuticals has just announced that vosoritide has just received a positive opinion from the Committee for Medicinal Products in Europe, which means marketing authorization is recommended. Further, it has just received temporary authorization in France.

Vosoritide is an injection of CNP taken once daily as a therapy for those with achondroplasia. Specifically, it is for patients who are at least 2 years old, and it is meant to be used until the growth plates close.

This therapy has already been given orphan drug designation from both the EMA and the FDA.


Achondroplasia is the most common type of skeletal dysplasia. Achondroplasia is caused by a change within the FGFR3 gene which is a negative regulatory for bone growth. It causes short stature characterized by endochondral ossification. This leads to short bones in the face, spine, long bones, and the base of the skull. Patients may also experience sleep apnea, foramen magnum compression, mid-face hypoplasia, bowed legs, spinal stenosis, sway within the low back, as well was recurrent ear infections.

As a result of these complications, patients often need surgery. For instance, patients may face invasive surgery to straighten their bowed legs or decompression of their spinal cord.

CHMP Opinion

The positive opinion was based on the data from the Phase 3 trial of this therapy which evaluated its safety and its efficacy. This trial was placebo-controlled, double-blind, and it further confirmed the positive findings from the Phase 2 investigation.

The Phase 2 trial was a dose-finding investigation which demonstrated that growth rates were maintained above baseline and above the expected growth velocity for patients who were untreated.

The Phase 3 trial included 121 patients between the age of 5 to 14. Besides the primary endpoint of growth velocity, a variety of secondary endpoints were evaluated. These included-

  • Height z-score
  • Joint geometry/limb and body proportionality
  • Biochemical assessments of bone growth
  • Biomarker assessments of bone growth
  • Radiological assessments  of bone growth
  • Quality of life
  • Functional independence
  • Developmental status

The study lasted for one full year. However, there is now an ongoing extension study which will last long term. This study has 119 patients enrolled. All participants in this trial are receiving 15 mcg/kg of vosoritide each day.

Looking Forward

The team expects that in Europe, there will be a final decision on vosoritide by the third quarter of this year.

If approved, this therapy would be the first therapy in Europe to receive approval for achondroplasia. It will be marketed under the name VOXZOGO.

You can read more about this investigative therapy for achondroplasia here.

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