Biomarin Pharmaceutical Inc. ((BMRN)) announced an update on their ongoing clinical study.
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BioMarin Pharmaceutical Inc. is conducting a long-term follow-up study titled ‘A Long-Term Follow-Up Study in Subjects With Severe Hemophilia A Who Received BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in a Prior BioMarin Clinical Trial.’ The study aims to evaluate the long-term safety and efficacy of BMN 270, a gene therapy for severe Hemophilia A, over a period extending beyond five years post-infusion. This research is significant for understanding the durability of the treatment’s effects.
The intervention being tested is BMN 270, an adeno-associated virus vector-mediated gene transfer of human Factor VIII. This treatment is designed to provide a long-lasting solution for patients with severe Hemophilia A by potentially reducing or eliminating the need for regular Factor VIII infusions.
The study is observational and follows a cohort model with a prospective time perspective. This means that participants who have previously received BMN 270 will be observed over time to gather data on the treatment’s long-term outcomes.
The study began on February 23, 2023, with the last update submitted on July 10, 2025. These dates are crucial as they mark the progress and ongoing nature of the study, which is currently enrolling by invitation.
The update from this study could have significant market implications, potentially boosting BioMarin’s stock performance and investor confidence if the long-term data supports the efficacy and safety of BMN 270. This could also influence the competitive landscape in the gene therapy market for Hemophilia A, where BioMarin is a key player.
The study is ongoing, and further details can be accessed on the ClinicalTrials portal.