After devastating regulatory setbacks last year, shares of uniQure and Replimune rose on the news that FDA Commissioner Marty Makary is leaving. But analysts don’t expect much relief, no matter who helms the agency.
Shares of UniQure and Replimune, two companies that received painful and unexpected rebukes from the FDA over the past year, climbed on the news that Commissioner Marty Makary has resigned.
But the departure may not mean much for these companies that have been struggling to move forward since the FDA flipped their regulatory worlds upside down.
UniQure’s shares have risen 23% to $28.44 apiece over the past five days. Rumors emerged of Makary’s potential firing on Friday, May 8.
Replimune, meanwhile, has climbed nearly 29% to $4.44, including an 8% rise as the markets opened this morning.
On Friday, BMO Capital Markets warned that Replimune’s situation is unlikely to change—even without Makary.
The FDA has rejected the company’s advanced melanoma drug RP1 twice. The first time was in July 2025, when the agency argued that the study used to underpin the application was not considered an “adequate and well-controlled clinical investigation,” Replimune said at the time.
Then-Center for Biologics Evaluation and Research (CBER) head Vinay Prasad was thought to have intervened at the 11th hour. It was later revealed, however, that former oncology head—and short-term Center for Drug Evaluation and Research director—Richard Pazdur had a hand in the rejection.
Then last month, a different team of FDA reviewers came to the same conclusion: a single-arm trial was not appropriate to support approval.
Makary himself defended the decision and stood by the scientists at the agency in an interview with CNBC that aired on May 5.
Replimune quickly announced that the biotech would have to cut jobs and scale back without timely accelerated approval of RP1.
“If Makary is removed as commissioner, we do not expect this meaningfully changes RP1’s second denial,” BMO wrote on Friday prior to the official resignation. The agency raised many concerns with the trial that would require a completely new study, the analysts said.
While Replimune’s shares are moving on the news of Makary’s departure, BMO said on Friday that RP1’s future is still in doubt.
“While REPL shares are reacting positively to this news, we expect a reversal on prior FDA CRLs for Replimune to be unlikely, even if Makary is replaced,” BMO wrote. “We’re not saying a revisit isn’t impossible, but we would not expect a quick resolution following a potential departure.”
UniQure, on the other hand, was preparing an application for the Huntington’s disease gene therapy AMT-130, which had recently recorded positive pivotal data that was heralded as a potential breakthrough for the intractable neurodegenerative disease.
But during a pre-BLA meeting, the FDA informed the company that the Phase 1/2 data would not be enough to support an application. This went against previous guidance that uniQure had received under the prior administration.
The FDA now wants uniQure to conduct a Phase 3 sham surgery-controlled trial—which would require patients to be anesthetized for 10 to 12 hours while a surgeon cuts through the skin and makes a burr hole in the skull.
In an attempt to overcome the FDA roadblock, uniQure has scheduled a type B meeting with the FDA this quarter to discuss a potential Phase 3 trial. At the same time, the company is preparing a regulatory package in the U.K. for the third quarter.
While both Replimune and uniQure may have a few good days at the market with Makary’s exit, analysts pointed to the larger ripple effects of the FDA chief’s departure.
“While near-term there are select names—most notably QURE and REPL—that could immediately benefit from the removal of regulatory headwinds tied to this particular leadership, over the more medium-term, we see today’s news as likely renewing an overhang at a time when biopharma had been performing relatively well,” RBC Capital Markets wrote Tuesday afternoon.
