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The US FDA’s Breakthrough Therapy designation is an expedited pathway for the development and review of medicinal products for treatment of serious conditions. This is applicable if sponsors can provide preliminary clinical evidence that indicates that the proposed drug is able to demonstrate significant and clear advantage compared to currently available therapy for a given clinically significant endpoint.

According to the US FDA a clinically significant endpoint, for a Breakthrough Therapy designation, is an endpoint that “measures an effect on irreversible morbidity or mortality (IMM) or on symptoms that represent serious consequences of the disease”.

How to request for a Breakthrough Therapy designation?

A drug that receives the Breakthrough Therapy designation can be considered for the Fast Track designation pathways and may be eligible to receive robust guidance for efficient product development from as early as Phase I stage.

Sponsors can request for a Breakthrough Therapy designation after the US FDA has reviewed the submitted data & preliminary clinical evidence, and thinks the drug development program can meet the Breakthrough Therapy designation requirements. Sponsors should submit the designation request before end-of-phase-2 meetings. The Agency will respond to designation requests within 60 days of request receipt.

What are the advantages of a Breakthrough Therapy designation?

The following are additional benefits that are associated with the Breakthrough Therapy designation and classified as appropriate per section 902 of the Food and Drug Administration Safety and Innovation Act (FDASIA):

More meetings between sponsors and US FDA review committees through drug development

Timely advice and communication with sponsor to ensure the development program can collect the necessary non-clinical and clinical data

Ensure clinical trial design is efficient and feasible and, where possible, exposes a smaller patient population to a potentially less effective therapy

Assign a project lead for FDA review team who also acts as a scientific liaison between various team members

Involve senior managers & experienced staff for cross-disciplinary review

Obtaining a Breakthrough Therapy designation from the US FDA presents sponsors with many challenges. For example, sponsors must demonstrate evidence from early-phase clinical trials that meets the US FDA’s stringent criteria for the designation, and proves the proposed therapy is superior to the existing treatment option. This demands extensive and comprehensive data collection & analysis, which necessitates sophisticated trial designs and endpoints.


The US FDA’s Breakthrough Therapy designation offers a vital expedited pathway for advancing treatments for serious medical conditions. The grant of the designation is depending on the sponsor’s ability to provide compelling preliminary clinical evidence to showcase the proposed product’s substantial advantage over existing therapies for critical clinical endpoints. The Breakthrough Therapy designation encourages increased collaboration between the Agency and sponsors which fosters a dynamic environment so that promising medical advancements can progress from “concept” to addressing serious conditions in patients.

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