Biomarin: Participant Diagnosed With Leukemia In Late-Stage Hemophilia Gene Therapy Trial
- In an SEC filing, BioMarin Pharmaceutical Inc said that, in August, a participant in its valoctocogene roxaparvovec (BMN 270) phase 3 study was diagnosed with B-cell acute lymphoblastic leukemia (B-ALL).
- Based on BioMarin's assessment of the case to date, including initial genetic testing of the leukemic cells, BioMarin believes at this time that this cancer is unrelated to BMN 270.
- In August, the hemophilia A gene therapy received conditional European approval.
- The overall rate of all cancers observed in all BMN 270 trial patients (2 in approximately 400 patient-years of observation) appears consistent with expected cancer rates in persons with Hemophilia.
- BioMarin said it started a comprehensive assessment of the case.
- Genomic analyses are underway, and BioMarin expects to confirm the absence of BMN 270 vector integration events contributing to leukemic growth.
- BioMarin submitted an IND safety report to the FDA consistent with the company's commitments to regulatory authorities.
- No authority has requested a hold on any trial at this time.
- The company says it remains on track to resubmit the marketing application for BMN 270 to the FDA by the end of this month.
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