From Concept to Regulatory Drug Approval: Lessons for Theranostics

The Journal of Nuclear Medicine

article

Marlon Perera¹  Michael J. Morris² 

[1] Urology Service, Department of Surgery, Memorial Sloan Kettering Cancer Center;Genitourinary Oncology Service, Department of Medicine, Memorial Sloan Kettering Cancer Center

关键词: theranostics;    PSMA;    FDA;    drug approval;    radium;    lutetium;   

DOI  :  10.2967/jnumed.121.263301

学科分类：医学（综合）

来源: Society of Nuclear Medicine

【 摘 要 】

Radiopharmaceutical therapy is an emerging treatment modality that has demonstrated increasing importance as a significant component in the treatment of cancer. Prostate cancer (PCa) remains one of the commonest solid-organ tumors and is associated with significant societal burdens. Despite significant disease heterogeneity, PCa remains an ideal candidate for radiopharmaceutical therapy because of the prolonged disease course, metastatic disease tropism, and sensitivity to radiation therapy. To date, advanced PCa remains one of the most successful arenas for the development and approval of radiopharmaceutical agents. In this review, we aim to summarize the complex processes required to obtain regulatory approval for a novel agent and highlight the limitations and hurdles specific to the approval of radiopharmaceutical agents. In advanced PCa, we outline the importance of a framework for trial design with respect to defining disease state and acceptable outcome measures—as recommended by the Prostate Cancer Clinical Trials Working Group (PCWG). Finally, using the principles mandated by the Food and Drug Administration approval process and the framework provided by the PCWG, we outline experience with the successful approval of the radiopharmaceutical agents 223Ra and 177Lu-PSMA-617.

【 授权许可】

CC BY