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BioMarin presented results of annualized growth velocity (AGV) in children with achondroplasia who started treatment during adolescence.

Data on 31 children under treatment for more than three years was recently presented at the Annual Meeting of the Pediatric Endocrinology Society. The average difference in AGV between treated and untreated children aged between 10 and 17 was 1.47 cm/year in girls and 1.71 cm/year in boys.

The improvement in annualized growth velocity in the group receiving the drug was maintained over a longer period of time compared to a population of average height, as opposed to an expected decline in AGV after the pubertal growth spurt (puberty).

With regard to the safety of the drug, the pharmaceutical company indicated that the results were consistent with previous studies in younger children and there was no evidence of negative effects on bone age or pubertal development.

Additional data demonstrates efficacy and gives preliminary signs of a positive impact on the health-related quality of life (HRQoL) of children with achondroplasia.

The results of another Phase 2 extension study, presented recently, showed that VOXZOGO had positive effects on growth in children who started treatment at less than 5 years of age. In children with more than 7 years of follow-up, the average increase in growth up to the age of 16 was 1.63 cm/year in boys and 1.33 cm/year in girls (lower than the initially indicated average increase of 1.56 cm/year).

In another Phase 3 study, the company reported evidence that VOXZOGO improved HRQoL in children, particularly aspects associated with functionality.

BioMarin presented information on the experimental administration of Voxzogo in idiopathic short stature, Noonan Syndrome and other growth-related conditions

Comment from Dr. Andrew Dauber:

"For the first time, we are seeing evidence that VOXZOGO can have a positive impact on growth in several dysplasias and growth-related conditions other than achondroplasia, including Noonan syndrome and ACAN deficiency which was presented today, as well as in hypochondroplasia which was recently shared. The safety and efficacy of VOXZOGO in achondroplasia are well established, we are encouraged by the new data to have an impact on an even wider group of children."
Andrew Dauber, M.D., Principal investigator and Chief of Endocrinology at Children's National Hospital, D.C.

At the same event, results were presented from a Phase 2 study of VOXZOGO in children aged 3-11 years with different growth-related genetic conditions, including Noonan syndrome and genetic mutations associated with idiopathic short stature, such as mutations in the ACAN gene, heterozygous NPR2 mutations and neurofibromatosis 1. The results provide evidence of an increase in ACV and standard deviation of height under the conditions studied and the safety results were consistent with the well-characterized safety profile of VOXZOGO.

For more detailed information, see BioMarin's full press release here.

In a recent publication in the journal Genetics in Medicine open, an extension study of 119 children with achondroplasia aged 5 to 18 years showed that the effect of Vosoritide on annualized growth velocity may be associated with gender. "The average difference in annualized growth velocity between treated and untreated children at all ages from 6 to 17 years was 1.46 cm/year, with average values of 0.61 cm/year in girls and 1.73 cm/year in boys.

The publications refer to average values and not final values per child.
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