Sen Zhuang, MD, PhD of Janssen Pharmaceuticals explains what a Breakthrough Therapy Designation means in terms of drug and clinical trial development.
A Breakthrough Therapy Designation is given to a drug by the U.S. Food and Drug Administration (FDA) if the agency determines, based on preliminary clinical evidence, that the drug will likely provide substantial improvement on at least one clinically significant endpoint compared to the current available therapy.
The designation will speed up the FDA review process of the drug, as well as allow the drug developers to meet with the FDA to provide guidance to further help expedite the development process (i.e., advise on clinical trail design).
Recently, the FDA granted Breakthrough Therapy Designation to JNJ-4528 for the treatment of multiple myeloma. JNJ-4528 is a B cell maturation antigen (BCMA)-directed chimeric antigen receptor T cell (CAR-T) therapy. Preliminary data from an ongoing Phase Ib/II clinical trial involving the drug to treat adults with relapsed or refractory multiple myeloma was instrumental in getting the Breakthrough Therapy Designation.
Multiple myeloma is a rare blood cancer affecting plasma cells. In 2019, more than 32,000 people will be diagnosed with multiple myeloma and about 13,000 will die from the disease in the United States. Numerous treatments are available for this condition, but relapse is common.
For more information about other rare cancers, visit checkrare.com/diseases/rare-cancers/