UniQure’s highly promising Huntington’s disease gene therapy BLA (biologics license application) was rejected by the FDA — because UniQure used an external control rather than a surgical sham control. Yet the latter would put control group patients at additional risk, making it ethically problematic. Hopefully some agreement will be reached which circumvents these issues! For now, it is an educational story to watch unfold.

Abi-Saab said during uniQure’s earnings call that he wouldn’t count on the four-year data altering the FDA’s decision.

“We don’t believe that there’s any reason we have today to believe that this will change the FDA’s position regarding the Phase 1/2 trials,” he told investors.

H.C. Wainwright struck a different tone, however, in the Monday note. “While the FDA appears to be enforcing a full sham surgery-controlled Phase 3 trial in Huntington’s, we believe an alternative path forward may be negotiated given the strong AMT-130 data generated to-date,” the analysts wrote. The 4-year data “should further inform the durability and magnitude of effect observed to date.”