RMAT, breakthrough designations and regulatory reversal

uniQure is a business that develops gene therapies. uniQure's AMT-130 is currently in the headlines. This podcast episode attempts to present the Huntington's Disease community's arguments for why the FDA should work collaboratively to design a palatable drug trial.

Last week the FDA told uniQure that AMT-130 would be subject to a double blind, Phase 3, randomized controlled study. While the scientific basis for that request is sound in most cases, the Huntington's Disease community argues that path is overly burdensome; perhaps impossible.

This episode cites:

Dr. Sung - https://www.youtube.com/watch?si=VhMQaFAZRvQA5NRj&t=62&v=mwEEJ91LeJY&feature=youtu.be

Sr. Sarah Tabrizi - https://www.youtube.com/watch?v=wKj1JRfJBRE&t=356s

Dr. Ed Wild - https://www.spreaker.com/episode/uniqure-update-with-dr-ed-wild--67900959

FDA papers - External Control Guidance https://www.fda.gov/media/122425/downloadUse of Bayesian Statistics - https://www.fda.gov/regulatory-information/search-fda-guidance-documents/use-bayesian-methodology-clinical-trials-drug-and-biological-products

Senate Committee on Aging - https://www.youtube.com/watch?v=yLm1SltbRxw&t=2702s

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