The FDA’s rare disease area is coming under increasing public and political scrutiny. There have now been 8 Wallstreet Journal articles as well as another article in Bloomberg calling for Marty Makary’s resignation.
Under his tenure, several promising drug candidates have been rejected. Replimmune and Uniqure both provided results on drug candidates that the FDA had previously indicated would be acceptable for approval. Both were rejected.
When Uniqure released its AMT-130 results, it received international attention among experts as a cure for Huntington’s disease. Its stock price shot up to $80, before falling because of Vinay Prasad’s (a Makary pick) decision to reject the drug and attack the trial design. Prasad is an advocate for a placebo control design that would require drilling holes into the heads of Huntington’s disease sufferers. This has been labelled unethical and infeasible by the medical community.
Prasad has since been sacked over the AMT-130 fall out. Makary will be too if he doesn’t turn this around.
Stock will bounce back to $80.