A biopharmaceutical company was seeking assistance to prepare a response to the FDA’s initial refusal of its orphan drug designation application for its novel CAR-T therapy intended for use in an orphan subset of a non-orphan condition.


After reviewing the FDA’s feedback, our expert FDA orphan drug consultants worked with the client to understand the available data and advise on a suitable justification for, and develop appropriate arguments to support, designation for the proposed subset. Our experts then searched the scientific literature, identifying credible supporting references and used these to prepare a robust response document based on sound medical, scientific and regulatory principles. Following client review of the response document, and submission to FDA, the Agency granted orphan drug status for the proposed orphan subset with no further questions.


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