FDA Rejects Promising Melanoma Drug RP1: Doctors Slam Decision as 'Shocking' and Life-Threatening

The U.S. Food and Drug Administration (FDA), under President Trump’s administration, has rejected a potentially life-saving melanoma treatment, sparking outrage from medical professionals.

On Wednesday, Secretary of Health and Human Services Robert F. Kennedy Jr. testified before Congress, asserting that he had no role in the FDA’s decision to withhold approval for Replimune’s drug RP1, which treats advanced melanoma. Instead, Kennedy shifted responsibility to FDA Commissioner Dr. Marty Makary.

“This decision comes out of FDA, and we trust the process there. And I’ve been told by Marty Makary that every panel that looked at that drug unanimously voted against it … because it does not appear to work.”

Kennedy’s claims were immediately disputed by oncologists, who argued in a Wall Street Journal op-ed that an initial FDA panel had actually approved RP1 before being overruled by the head of biologics, Dr. Vinay Prasad.

The oncologists also challenged Kennedy’s assertion from a House hearing that RP1’s clinical trial was flawed because all patients received chemotherapy. In reality, none of the trial participants received chemotherapy—they were given a different form of immunotherapy.

Dr. Anna Pavlick, a melanoma researcher involved in the trial, told the Journal:

“Honestly, there was no doubt in our minds whatsoever when we completed this study and we saw the results, that this was going to be approved as a wonderful alternative for our patients because they have no options. I have patients who have been treated with this drug that are still alive today who would otherwise be dead.”

Dr. Eric Whitman of the Atlantic Health System Cancer Care echoed Pavlick’s sentiments:

“When you talk to the melanoma experts, people who treat lots and lots of melanoma patients like myself, it’s obvious that this is beneficial to patients and it’s saving lives or it has potential to save lives. The community of patients and doctors don’t understand the reasoning” for the FDA’s rejection.

The rejection of RP1 follows a pattern of controversial decisions under Kennedy’s leadership at HHS, including blocking a CDC study that found the Covid-19 vaccine significantly reduced emergency room visits and hospitalizations last winter. With RP1 showing strong potential against a deadly cancer, its rejection has intensified concerns about the Trump administration’s approach to public health and cancer treatment.