How Replimune’s Drug Got Third Chance After White House Intervention
Company representatives met with officials in early May and by month’s end, the FDA was planning to review the melanoma medication again
- The White House pushed health officials to re-examine Replimune’s melanoma drug after FDA rejections, leading to a resubmission agreement.
- The FDA had twice rejected the drug, citing insufficient data from a trial without a comparison group and a hard-to-interpret patient mix.
- Replimune will resubmit its drug application with new data, causing its shares to rise more than 80% Friday.
The White House pushed health officials to re-examine the Food and Drug Administration’s repeated rejections of an advanced melanoma medication from Replimune Group REPL 85.68%increase; green up pointing triangle, people familiar with the matter said.
The FDA had declined to approve Replimune’s treatment in July last year and then again in April, saying the data from the trial, in which all patients received the experimental drug with no comparison group, fell short of proving to the agency that it worked. The FDA also said the mix of patients enrolled made the results hard to interpret.
After its application was rejected for the second time, Replimune representatives met with the White House in early May, arguing the rejections didn’t match up with the Trump administration’s desire to help terminally ill patients.
White House staff later asked health officials to re-examine the drug’s prior rejections to determine whether there was any wrongdoing in the process, the people said. Health department lawyers compiled a report about the drug review and shared the results with White House officials, the people said. The report found that the FDA had made no procedural errors in how it handled the earlier rejections, one of the people said.
Replimune said Friday it had reached an agreement with the FDA to resubmit its drug for a third attempt at approval.
The administration didn’t instruct health officials to approve Replimune’s drug, and the White House has since handed the reins to the FDA, people familiar with the events said.
The White House declined to comment.
The stark reversal comes less than three weeks after the resignation of FDA Commissioner Marty Makary. He had clashed with top officials in the Department of Health and Human Services and the White House over many matters, including vaping and tobacco policy. Rare-disease patients and their advocates criticized Makary for not being flexible enough when it comes to evidence to prove treatments for their ailments work.
Kyle Diamantas, a lawyer who was running food regulation at the agency, has since been named acting commissioner.
Some industry representatives criticized Makary for the FDA’s Replimune rejections, adding pressure when he was already on thin ice with administration officials over vaping policy and ongoing management clashes.
While still head of the agency, Makary publicly defended the rejections of Replimune’s medication. “If your drug works, it’s going to get approved,” he said in an interview with CNBC.
Replimune had accused the FDA of being inconsistent in its prior treatment of the drug, with different teams reviewing its drug each time.
Now, Replimune has another shot at getting its experimental treatment approved. The company will be presenting health officials with data that it hadn’t previously shared with the agency, people familiar with the plan said.
Replimune said it plans to resubmit the application in the coming days. Its shares were up more than 80% Friday.
About a third of patients with treatment-resistant melanoma saw their tumors shrink after taking Replimune’s drug combined with a second cancer drug, Bristol-Myers Squibb’s Opdivo.
More than 110,000 people in the U.S. are expected to be diagnosed with melanoma this year, the National Cancer Institute estimates, and more than 8,000 people with advanced disease are expected to die nationwide.