In the first part of his interview, Dr Jenkins shared with Biotech Strategy Blog the FDA perspective on what constitutes a breakthrough drug? Given he is one of the senior managers at the FDA and sits on the committee that decides whether to grant or deny a company’s breakthrough therapy request, his opinion counts.
In the second and final part of the interview, Dr Jenkins discusses the advantages and benefits of the Breakthrough Therapy designation, as well as some of the challenges the agency faces in administering it.
Receiving a breakthrough designation is no guarantee of FDA approval. Drisapersen (GSK/Prosena), a drug for the treatment of Duchenne muscular dystrophy failed a phase III trial in September, despite having received a breakthrough therapy designation in late June.
In this respect, the breakthrough therapy designation is no different from other expedited pathways such as accelerated approval, fast-track or priority review: you still have to generate clinical trial data from a registration trial that supports the initial promise shown.
What then, does the breakthrough therapy designation mean for cancer drug development? Subscribers can read below the second part of the interview with John Jenkins MD, Director, Office of New Drugs, Center for Drug Evaluation and Research at the FDA: