FDA’s uniQure reversal unlikely to impact Regenxbio’s programs, says Stifel

Stifel analyst Annabel Samimy says that the news regarding FDA’s reversal on the approvability of uniQure’s (QURE) AMT-130 on Phase 1/2 data on the surface injects another layer of uncertainty into the workings of FDA and their receptivity to gene therapy more specifically. The natural response is to draw parallels for the broader GTx space, which encompasses Regenxbio’s (RGNX) rare disease programs. Stifel says that while it is not surprised by the pressure on Regenxbio’s shares, it stands by the quality of the program designs, the data generated to date, and the company’s modernized, scalable, and reproducible manufacturing capacity, of which FDA already completed both the required PLI and BIMO inspections with NO observations/issues, a relatively unprecedented outcome in the GTx space. The firm has a Buy rating on Regenxbio.