FDA’s N-of-One call to action for academics

FDA’s new N-of-One pathway would enable academics to tailor-make therapies for single patients

CDER Director Janet Woodcock invites academic researchers to create therapies for individual patients using FDA’s new N-of-One pathway, but she emphasizes the program will not allow companies to skirt traditional drug development.

The advent of gene therapy and antisense oligonucleotides has opened the door to tailoring therapies to specific mutations found in single patients. The lack of commercial incentive, however, means these are almost never pursued.

In a rare exception, Timothy Yu and colleagues at Harvard Medical School designed a splice-modulating antisense oligonucleotide to treat a single patient with a unique genotype that causes a rare, fatal neurodegenerative disease. Results from the N-of-One study were published in the October edition of the New England Journal of Medicine.

Based on Yu’s experience and findings, Woodcock and CBER director Peter Marks unveiled an N-of One drug development pathway in an Editorial published in the same NEJM issue.

The pathway is intended for academic researchers who treat patients and want to develop an individualized therapy similar to the one developed by Yu,

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