biotechnology

FDA shift under acting commissioner lifts rare-disease biotech stocks

• The FDA under acting Commissioner Kyle Diamantas has approved Replimune's melanoma treatment and cleared uniQure to file its Huntington's disease gene therapy, reversing earlier FDA decisions on both drugs.
• The shift reverses a stance taken in the first year of the current Trump administration, when FDA officials grew skeptical of approving drugs tested in tiny patient groups, sometimes without the usual features of large trials such as placebo controls.
• uniQure's therapy could reach an estimated 40,000 U.S. patients with Huntington's disease, a neurodegenerative disorder with no approved treatments shown to slow its progression.
• Analyst Joseph Thome of TD Cowen said the odds of accelerated approval for uniQure are "skewing positive" because the FDA is returning to its "old body language."
• Regenxbio illustrates the limits of the reversal: after the FDA cleared a resubmission in June, the agency placed the company's gene therapy on clinical hold in August when spinal scans turned up abnormalities in trial participants.