The Breakthrough Designation Can be Withdrawn or Rescinded by FDA 
Posted on: Thursday, July 2, 2015

Since the launch of the Breakthrough (BT) designation program by FDA in July 2012, there have been more than 200 requests for this designation just to CDER, along with several additional to CBER. FDA granted about one-third of the requests, and rejected the other applications for either lack of clinical evidence, safety issues, or no significant improvement over existing therapies. Once a company successfully gets the BT designation, it is cause of major uptick in the value of the product due to scientific validity of the program and potential faster market approval. Some analysts have set the valuation of BT designation to hundreds of millions of dollars. So, obviously this designation is very precious. However, it is not a permanent designation. Under certain conditions, FDA may ask the sponsor to voluntarily withdraw the BT designation or FDA can rescind the BT designation. So far, there have been 2 cases of withdrawal of BT designation after granting by FDA and 4 cases where FDA rescinded the BT designation. This could happen because in the opinion of the FDA the company was too slow to develop the BT designated drug, i.e., the clinical study was delayed beyond reasonable or the drug manufacturing was not ramped up to meet the clinical trial and market needs. Alternatively, the BT designation can be rescinded if FDA approves another new drug for the same disease making the BT designation is no longer deemed necessary. It is important for companies to not get too relaxed upon receiving high value incentives from FDA and pursue the incentive relentlessly. One must keep in constant communication with FDA to address any FDA comments promptly and keep the momentum of getting approval as quickly as possible by completing the clinical development program and filing the NDA/BLA at the earliest, lest FDA rescind designation due to change in the patient need due to availability of newer competing therapies.

Expert Opinion: Mukesh Kumar
VP, RA, Amarex Clinical Research