FDA expands indication for vosoritide to include children of all ages with achondroplasia
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Key takeaways:
- The FDA has expanded its accelerated approval for vosoritide to include children of all ages with achondroplasia.
- Vosoritide improved mean height during an open-label phase 2 extension study.
The FDA has expanded its accelerated approval of an injectable therapy for treating achondroplasia to include children of all ages, according to a press release.
The FDA approved a new indication for vosoritide (Voxzogo, BioMarin Pharmaceutical Inc.) to include children of all ages with achondroplasia with open epiphyses, or open growth plates. As Healio previously reported, vosoritide was the first drug approved by the FDA to treat achondroplasia and open epiphyses in November 2021. The agent was originally granted accelerated approval for children aged 5 years and older.
The expanded indication was based on findings from a double-blind, placebo-controlled phase 2 trial analyzing the efficacy and safety of vosoritide among children aged 5 years and younger. According to the press release, results from the phase 2 trial along with findings from a phase 3 trial of children aged 5 years and older have established that vosoritide improves linear growth for children of all ages with achondroplasia and open epiphyses. The safety profile of vosoritide for children aged younger than 5 years was similar to what was observed in children aged 5 years and older.
Findings from an open-label, long-term phase 2 extension study were presented at the 2023 European Society for Pediatric Endocrinology Meeting in September. Children aged 2 years and older who received vosoritide over a 4-year period had mean height z score improve by 1.1 to 1.4 standard deviations and a mean height gain of 6.3 to 7.8 cm compared with children of the same age and sex who did not receive treatment. Children aged less than 2 years who received vosoritide over a 3-year period had a mean height z score improvement of 0.8 to 1 standard deviations and a mean height increase of 3.5 to 3.9 cm.
"We are pleased that Voxzogo is now available for children of all ages with achondroplasia," Hank Fuchs, MD, president of worldwide research and development at BioMarin, said in a press release. "We are grateful for the collaboration of the achondroplasia community, physicians and the children and their families who have played a crucial role in advancing this clinical program. We also look forward to further understanding the potential role of Voxzogo in other genetic short stature conditions, including hypochondroplasia."
The new indication from the FDA is under accelerated approval based on an improvement in annualized growth velocity. Continued approval is contingent upon verification and description of clinical benefits in confirmatory trials, which BioMarin plans to fulfill by comparing data from ongoing open-label extension studies to available natural history.
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