NHF Requests Risk Evaluation and Mitigation Strategy for Pending Gene Therapy Treatments

Read a citizen’s petition that was formally submitted to the Food and Drug Administration by NHF.

On July 1, the National Hemophilia Foundation (NHF) submitted a citizen’s petition to the Food and Drug Administration (FDA) in relation to the pending approval of two gene therapy treatments. The intent of the requests in this petition are to ensure the health and well-being of people with hemophilia who receive a gene therapy product.

In the document, NHF requested a risk evaluation and mitigation strategy (REMS) for both valoctocogene roxaparvovec, a BioMarin investigational gene therapy under regulatory review for the treatment of severe hemophilia A, and etranacogene dezaparvovec, a CSL Behring investigational gene therapy currently under review for the treatment of hemophilia B.

NHF places the safety of the community first and foremost, which is why the request has been made. According to Leonard Valentino, MD, NHF’s President and CEO, “We at the National Hemophilia Foundation look forward to the innovation and improved outcomes that gene therapy may offer to people with hemophilia many uncertainties with these products still remain, which we may not fully appreciate for years to come. We must ensure that the FDA implements a rigorous safety program in an effort to protect our community.”  Gene therapy, while highly promising, is a treatment decision that members of the community should make in close consultation with their medical team and families to address the long-term risks and benefits. Before being heralded as a potentially life-changing treatment, gene therapy should be evaluated with safety outcomes in mind by the regulatory authorities.

NHF is thrilled by the possibility of the benefits that gene therapy may bring, but moves forward with cautious optimism with the community in mind. NHF will continue to partner with the bio-pharmaceutical companies, as well as researchers, health care providers, and others as gene therapy continues to emerge as a treatment option. NHF will work with all partners to encourage truthful, timely, transparent communication about all safety concerns.

Read NHF’s citizen petition here. The FDA is expected to respond to NHF’s request within the next 200 days. To learn more about submitting your own citizen petition, click here.

Source: National Hemophilia Foundation

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