On Saturday, September 10, 2022, BioMarin announced that a hemophilia A patient participating in a gene therapy clinical trial – in which they had been treated with BMN270 – has been diagnosed with pre B cell acute lymphoblastic leukemia. 

After analyses, BioMarin reports that there is no evidence of AAV DNA sequences as causative of the tumor genesis. You can read the statement from BioMarin here.

As a patient advocacy organization, the National Hemophilia Foundation (NHF) is dedicated to the safety of the inheritable blood disorders community. Pharmacovigilance will always be a top priority to assess, understand, and prevent any adverse effects of treatments for the community.

On July 1, 2022 a citizens petition was submitted to the Food and Drug Administration by NHF in relation to the pending approval of two gene therapy treatments. The intent of the requests in the petition is to ensure the health and well-being of people with hemophilia who receive a gene therapy product. NHF is optimistic for the potential benefits that gene therapy may bring, but moves forward with caution and with the community’s safety in mind.

NHF is also communicating with our international partners (WFH, EHC) regarding our safety related concerns related to this incident.