BioMarin Says One Participant Diagnosed With Leukemia In Late-Stage Hemophilia Gene Therapy Trial

In an SEC filing, BioMarin Pharmaceutical Inc BMRN 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.
Related: BioMarin Lifts Annual Guidance On Better Than Expected Q2 Earnings.
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.
Price Action: BMRN shares are down 3.15% at $90.00 on the last check Monday.