Biomarin Pharmaceutical Inc. ((BMRN)) announced an update on their ongoing clinical study.
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BioMarin Pharmaceutical Inc. is conducting a Phase 2, randomized, controlled, multicenter study titled ‘A Phase 2 Study of Vosoritide in Children with Idiopathic Short Stature.’ The study aims to evaluate the effects of multiple doses of vosoritide and compare its therapeutic dose to human growth hormone (hGH) in children with idiopathic short stature (ISS). This research is significant as it explores potential new treatments for ISS, which could offer alternatives to existing therapies.
The study tests vosoritide, an experimental drug administered via daily injection, in three dosage levels (low, medium, and high). A placebo and hGH are used as comparators to assess the efficacy of vosoritide.
The study design is interventional with a randomized, parallel assignment model. It employs quadruple masking, meaning participants, care providers, investigators, and outcomes assessors are blinded to the treatment allocations. The primary purpose is treatment-focused.
The study began on April 19, 2024, with a recent update submitted on January 17, 2025. These dates are crucial as they indicate the study’s progression and ongoing recruitment status, which is currently active.
This clinical update could influence BioMarin’s stock performance by potentially boosting investor confidence if the results show positive outcomes. The study could also impact the competitive landscape in the ISS treatment market, where alternative therapies are in demand.
The study is ongoing, and further details can be accessed on the ClinicalTrials portal.