VoxzogoAchondroplasia (dwarfism) in children
New molecular entity
Development and Regulatory status
Phase III Clinical Trials
Recommended for approval (Positive opinion)
Jun 21Recommended for EU approval by CHMP – the full indication is “Voxzogo is indicated for the treatment of achondroplasia in patients 2 years of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing. Voxzogo will be available as 0.4mg, 0.56mg and 1.2mg vials of powder and a solvent for solution for injection .
Nov 20NICE TA scoping exercise delayed until 2023, assume UK launch is also .
Nov 20FDA accepts NDA for vosoritide and a decision is expected by August 2021 .
Aug 20Biomarin has submitted a New Drug Application to the U.S. Food and Drug Administration (FDA) for vosoritide for children with achondroplasia . 24/08/2020 12:57:16 : Susan Carr
Jul 20Filed in EU via centralised procedure for treatment of achondroplasia .
Feb 20BioMarin is planning to file in 2020 .
Dec 19Based on data from the PIII trial, the company plans to meet with health authorities in H1 2020 to discuss plans for submitting marketing applications. 
The FGFR3 gene mutation interferes with how chondrocytes multiply and line up. This C-type natriuretic peptide stimulant initiates intracellular signals that inhibit the overactive FGFR3 pathway to normalise bone growth .
Achondroplasia is the most commonly occurring abnormality of bone growth (skeletal dysplasia), occurring in approximately 1 in 20,000-30,000 live births. It occurs as a result of a spontaneous genetic mutation in ~80% of pts and is inherited in ~20%. It appears to affect males and females in equal numbers.
Achondroplasia (dwarfism) in children
To be confirmed
Trial or other data
Sep 20PIII study (NCT03197766; n=121) found once-daily subcutaneous vosoritide was associated with increased growth versus placebo in children with achondroplasia (adjusted mean difference in annualised growth velocity of 1.57cm/year in favour of vosoritide; 95% CI 1.22–1.93]; p<0.0001) .
Dec 19Positive final results announced from randomised, double-blind, placebo-controlled PIII trial (NCT03197766)evaluating the efficacy and safety of vosoritide as a once daily s.c. 15ug/kg/day injection in 121 children aged 5-15. The placebo-adjusted change from baseline in growth velocity after 1 yr (primary endpoint) was 1.6 cm/yr (p<0.0001).
Jun 19PII open label extension trial initiated to evaluate the safety and efficacy of vosoritide in ~70 children in the US with achondroplasia (NCT03989947). Data are expected in Q3 2026. [3,7]
Jun 19Positive safety and efficacy data from PII trial (NCT03583697). The treated cohort gained a mean of 5.7cm of cumulative height over predicted height. The annualised growth velocity increased from baseline in all cohorts during each 12-month interval by 1.10 to 2.34 cm/year through 42 months. Vsoritide was safe and well tolerated though mild and transient injection-site reactions were reported. Serious adverse events occurred in 4 pts (grade 3 obstructive sleep apnea, thyroglossal cyst, syrinx and grade 1 tonsillar hypertrophy).
May 18PII randomised, placebo-controlled trial (NCT03583697) initiated to assess the efficacy, safety and tolerability of vosoritide in ~ 70 children in the US, Australia and the UK aged 0-5 with achondroplasia.[3,8]
Dec 17A PIII open label long-term extension trial is initiated to evaluate the safety and efficacy of the 15 µg/kg/day dose of vosoritide in children in Australia who participated in a previous PIII study (NCT03424018 - for this long term extension study). Data are expected in Q3 2024. [3,6]
Dec 16Randomised, placebo-controlled PIII trial (NCT03197766) initiated to evaluate the safety and efficacy of vosoritide in 110 children (aged 5-14) with achondroplasia ages 5-14 in Australia, Spain and the UK. The primary endpoint is the change in growth velocity from baseline over 1 year vs. placebo.[3,5]