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Drug - Voxzogo™ (vosoritide) [BioMarin Pharmaceutical, Inc.]

April 2022

Therapeutic area - Growth Factors

Initial approval criteria

  • Patient is at least 5 years of age AND
  • Patient has a definitive diagnosis of achondroplasia as confirmed by one of the following: 
    • Clinical (e.g., proximal shortening of arms, large head, narrow chest, short fingers) and radiographic (e.g., ilia and horizonal acetabula, narrow sacrosciatic notch, proximal radiolucency of the femurs, generalized metaphyseal abnormality, decreasing interpedicular distance caudally) features consistent with the disorder OR 
    • Identification of a heterozygous pathogenic variant in the FGFR3 gene (e.g., 1138G>A and 1138G>C being the 2 most common), by molecular genetic testing AND 
  • Other causes of achondroplasia or short stature have been ruled out (e.g., malnutrition, hypothyroidism, hypocortisolism, hypochondroplasia, thanatophoric dysplasia, SADDAN syndrome, homozygous achondroplasia [excludes approved labeled indication]) AND 
  • Patient does not have closure of epiphyses AND 
  • Patient body weight, growth, and physical development will be measured at baseline and monitored throughout therapy AND 
  • Therapy will not be used in combination with growth hormone (e.g., somatropin), growth hormone analogs (e.g., somapacitan), or insulin-like growth factor (IGF-1) (e.g., mecasermin) AND 
  • Patient has an estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73 m2 AND 
  • Patient has not received limb-lengthening surgery within the previous 18 months

Renewal criteria

  • Patient continues to meet above criteria AND 
  • Patients does not have closure of epiphyses AND 
  • Patient has not had treatment-limiting toxicity (e.g., severe hypotension) AND 
  • Patient has shown a beneficial response to treatment as evidenced by the following:
    • Improvement in height compared to pre-treatment baseline AND
    • Improvement in growth velocity compared to pre-treatment baseline

Quantity limits

  • 0.4 mg single-dose vial for reconstitution:  1 vial daily 
  • 0.56 mg single-dose vial for reconstitution:  1 vial daily 
  • 1.2 mg single-dose vial for reconstitution:  1 vial daily


Voxzogo is a C type natriuretic peptide (CNP) analog indicated to increase linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).


MHCP Provider Resource Center 651-431-2700 or 1-800-366-5411

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