In 2019, a measure was approved by the Brazilian Health Regulatory Agency (Anvisa) to regulate gene therapy as a drug and to establish specific criteria for clinical studies using gene therapy. Because the measure clarified the approval process for gene therapy research, it is expected to increase gene therapy research being conducted. Additional legislation is expected to be finalized later this year, which will include specific regulations outlining how to file applications for registration of new gene therapy drugs.
Gene therapies are regulated by Anvisa, which is responsible for clinical trial oversight, approval, and inspections for drugs registered in Brazil. Prior to 2019, it was unclear whether gene therapy products would be regulated as drugs or as human tissues. In 2019, the agency approved RDC #260, which establishes gene therapy as a drug and created the regulatory framework for advanced cell therapies including gene therapy. No gene therapies have been approved or registered yet.
- Prostate cancer in mice: In 2019, researchers at the São Paulo State Cancer Institute (ICESP) used gene therapy to destroy tumor cells upon injection into mice with prostate cancer and to make tumor cells more sensitive to chemotherapy drugs. More studies will be conducted before clinical trials in humans begin.
- Fungal infections: Researchers used stem cell therapy to treat patients with a genetic mutation that causes them to be more susceptible to fungal infections.
- Heart disease: Researchers conducted clinical trials to test gene therapy to reduce symptoms of refractory angina, a coronary artery disease.
- Head and neck cancer: Researchers at the University of Sao Paulo tested gene therapy as a treatment for advanced head and neck carcinoma.
2019: Anvisa approves RDC #260, which establishes specific criteria for gene therapy research and development and opens the door for more gene therapy research.
2018: Anvisa approves RDC #214, which establishes regulations for clinical research using human cells.
2012: The National Health Council passes CNS Resolution #466, which provides a general framework for human research ethics.
1995: Brazil passes Law No. 8.974, which establishes safety and inspection requirements for genetic engineering in agriculture and humans. The aim is to protect human, animal and plant health as well as the environment. It establishes which manipulation methods would be prohibited.
- Genetic Literacy Project’s FAQ on gene editing
- Library of Congress summary of Brazilian gene regulations includes detailed analysis of the country’s evolving biosafety laws and liabilities