uniQure's Huntington's Gene Therapy Faces FDA Study Requirement

Stifel says that it can see why uniQure's (QURE) shares are lower on CNBC comments from FDA commissioner Marty Makary, where he was interviewed about FDA's approach to rare disease. Makary was defensive of FDA's approach broadly, as he appeared to maybe refer to the AMT-130 program as an example of something his FDA didn't get behind. To be clear, his specific commentary might/might not have been referring to AMT-130 since uniQure never received an actual CRL, the firm notes. Some seem to think Makary was referring to Regenxbio's (RGNX) RGX-121, but this doesn't really make sense to Stifel since he describes a drug that didn't beat a control arm, and the Regenxbio study in Hunter was single-arm. Specific details aside, what's also notable is his steadfast defense of FDA which is discouraging for those hoping that the agency can be convinced to reconsider recent negative decisions, in the absence of specific political pressure, argues the firm.