BMRN stock falls after leukemia case in late-stage trial for hemophilia drug (NASDAQ:BMRN)

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Commercial-stage biotech BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced Monday that investigations are underway after a patient in its Phase 3 trial for its Hemophilia A candidate BMN 270 was diagnosed with B-cell acute lymphoblastic leukemia (B-ALL).

Citing initial studies, including genetic testing of leukemic cells, BMRN said that the cancer is unrelated to BMN 270, a gene therapy based on an adeno-associated virus (AAV) vector called AAV5.

The company is conducting additional genomic analysis to confirm if the leukemic growth was unrelated to BMN 270 vector integration and to provide further details on the genetic factors that caused cancer.

However, BMRN submitted an IND safety report to the FDA and shared the findings with the global health officials on September 02.

No regulatory agency has requested a clinical hold on any of the trials. The company’s independent Data Safety Monitoring Board has determined that the event did not require modifications to the BMRN’s ongoing studies.

The company remains on track to resubmit the Biologics License Application for BMN 270 to the FDA by the end of September.

The event comes less than a month after European regulators granted conditional marketing authorization for BMN 270, also known as valoctocogene roxaparvovec and Roctavian in commercial terms, as a treatment for adults with severe hemophilia A.

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