Biotech Strategy Blog

Commentary on Science, Innovation & New Products with a focus on Oncology, Hematology & Cancer Immunotherapy

Posts tagged ‘FDA Breakthrough Therapy’

IFDA Logon 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:

Earlier this month, Janssen/Pharmacyclics announced they had submitted a New Drug Application (NDA) for Food & Drug Administration (FDA) approval of ibrutinib, an oral Bruton’s tyrosine kinase inhibitor (BTK) in chronic lymphocytic leukemia (CLL) for the treatment of patients with a deletion of the short arm of chromosome 17 (del17p). Here’s a link to July 10 press release.

The company have requested Priority review; approval later this year or in early 2014 is highly likely given that the agent has also been designated a Breakthrough Therapy by the FDA.

This is great news for CLL patients!

CLL is an incurable disease. It is the most common leukemia in the United States with 15,500 new diagnoses a year.

Chromosomal abnormalities are fairly common in CLL and predict both time to first treatment and overall survival i.e. how long someone will live. Sadly, those with a 17p deletion have the worst outcome and a poor prognosis.

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