Vosoritide boosts annual height velocity with multiple genetic causes of short stature
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Key takeaways:
- Children with five genetic causes of short stature had height velocity increases of 3 cm or more with vosoritide.
- Increases in genetic testing for short stature may lead to precision treatment in the future.
Vosoritide was associated with increased annualized height velocity for children with five different genetic causes of short stature, according to study findings.
As Healio previously reported, vosoritide (Voxzogo, BioMarin Pharmaceutical) boosted annualized height velocity among children with hypochondroplasia in a phase 2 trial. New data from the same trial presented at the Pediatric Endocrine Society annual meeting revealed the medication also was associated with increased annualized heigh velocity among children with other causes of short stature.
“Preliminarily, we saw an increase in growth velocity of over 3 cm per year across all of the subgroups,” Andrew Dauber, MD, MMSc, chief of endocrinology at Children’s National Hospital in Washington, D.C., and principal investigator of the study, told Healio. “That’s really positive, that’s a robust growth response and really proves the principle that if you target this pathway these disorders are directly affecting, you can improve growth.”
Researchers recruited boys aged 3 to 10 years and girls aged 3 to 9 years who had a genetic variant for short stature, a height standard deviation score of less than –2.25 and no growth hormone deficiency. After a 6-month observation period where baseline annualized growth velocity was obtained, participants received 15 g/kg vosoritide once a day for 1 year. Annualized growth velocity and height standard deviation score were obtained at 6 months and 1 year. Adverse events were collected. The data presented at the Pediatric Endocrine Society included 30 children who had a genetic variant that was not hypochondroplasia (mean age, 7.1 years; 73% boys).
Of the children in the analysis, 12 had aggrecan deficiency, eight had Noonan syndrome, seven had a heterozygous NPR2 mutation, two had neurofibromatosis type 1 and one had Costello syndrome.
Mean annualized growth velocity increased from 4.5 cm per year at baseline to 8.5 cm per year at 6 months and 8.1 cm per year at 1 year. From the start of treatment to 1 year, mean height standard deviation increased for all genetic causes.
No participants discontinued vosoritide due to adverse events. Injection site reactions were reported by 33% of participants. Two grade 3 adverse events were reported, both events were occurrences of genu valgum. Both children recovered with surgical correction and ongoing treatment. Dauber said both participants with the condition continued to receive vosoritide.
Vosoritide could be a game changer down the road for children with various causes of short stature, according to Dauber. Some of the genetic causes in the study do not have an FDA-approved treatment, he said, and he believes that genetic testing to identify the specific cause of short stature for children could allow a targeted therapy approach in the future.
“Step one is promoting the appropriate genetic testing and access to diagnostic testing to identify these patients,” Dauber said. “After this, we really need to do a larger, well-controlled trial, either placebo-controlled or comparison to growth hormone and placebo, to show in these conditions what really is the effect size, how long will it last and is this a lasting effect over multiple years. But preliminarily, it looks really encouraging.”
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Galetaki D, et al. Oral abstract 6512. Presented at: Pediatric Endocrine Society Annual Meeting; May 2-5, 2024; Chicago.
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