Regulatory strategy relies heavily on experts with understanding of regulatory agency nuances and changing patterns and regulations. Since the FDA’s introduction of breakthrough-therapy designations in 2012, much emphasis has been placed on guidelines and qualifications for breakthrough-therapy status and how it compares to its earlier predecessors of expedited agency review including orphan designation, breakthrough status, and fast-track status.


A pharmaceutical subsidiary of an international organization with over $10 billion in annual revenue was seeking breakthrough-therapy designation from the FDA for their novel central nervous system (CNS) compound. Receiving breakthrough-therapy designation expedites the drug approval process, saving time and money for the client, and introducing life-saving treatment to patients as quickly as possible. At the time of submission, the FDA had awarded breakthrough-therapy status to only 64 drugs, none of which were CNS compounds. A lack of precedent made this regulatory engagement a unique challenge.


The Venebio team for this project was selected based on their extensive experience in regulatory submissions, medical writing, and clinical trials. Venebio prepared a submission-ready Cover Letter and Designation Request Package, and facilitated submission and interaction with the FDA. An emphasis on the compound’s potential to address a life-threatening disease and its lack of market attention was the cornerstone of the request. A review of the proposed indication and dosing guidance, current regulatory status, and current clinical data was included in the package.


Soon after submission to the FDA, the client received news that their compound had become the first CNS drug to be granted breakthrough-therapy status. The favorable outcome saved our client a significant amount of time by expediting the market approval process, resulting in a direct and substantial cost savings by allowing for a quicker path to market and resulting revenue. The historic award also provided a positive boost for the client’s public image.

How Venebio addressed the challenge

Regulatory engagement of any kind requires a diverse and nuanced skillset with an understanding of regulatory history, evolving current practices, clinical strategy and scientific writing. Venebio’s in-depth understanding of regulatory requirements and submissions and experience in drafting agency-facing reports was an essential part of this first-of-its kind application.

Drug/Device Development & Safety