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Certain medicines have been given a new type of status by the Food and Drug Administration (FDA), better known as Breakthrough Therapy Designations.

By Matthew Wood on Feb 15, 2013

To expand, Breakthrough Therapy Designation is intended to accelerate both the development and review time for a potential new drug to treat a serious or life-threatening disease or condition where preliminary clinical evidence indicates that the drug may demonstrate significant improvement over existing therapies observed early in clinical development.

The designation of a drug as a Breakthrough Therapy was enacted as part of the 2012 FDA Safety and Innovation Act. In accordance with the act, this designiation will allow for the following: more meetings to take place during the development process, potentially reduce the number of patients to enroll in further clinical trials, and (as a result) shorten the length of those trials.

In essence government believes that there are some medicines that have proven to possess significant promise in offering new hope to patients during early study phases, thereby having the opportunity to arrive on the market without having to go through the traditional three-phase development plan.

However, some of you would agree when Janet Woodcock of the FDA’s Centre for Drug Evaluation and Research warned that the FDA is more likely to align themselves with a proven developer who has a strong reputation and an even stronger knowledge base of the disease in question.

Woodcock continued in saying, “Breakthrough status means the companies will have closer communication with higher ranking FDA personnel to move drugs for serious diseases to market more quickly, potentially with data from an expanded phase 1 trial. It’s probably going to take some time for this program to become established in the USA but there’s robust interest.”

At present, three drugs have won Breakthrough Therapy Designation status and can perhaps serve as appropriate guidelines as to what the FDA are looking for. Janssen Biotech Inc. and Pharmacyclic’s Ibrutinib for B-cell malignancies, and Vertex’s Kalydeco and VX-809 (being studied as a combination treatment) for cystic fibrosis have been granted status.

“This is an historic moment in oncology,” said Pharmacyclics CEO and Chairman of the Board Bob Duggan, “We are truly honored to have received this Breakthrough Designation and are pleased for patients and clinicians with the FDA’s decision to expedite the development of Ibrutinib.”