New FDA-Approved Therapy Offers Hope for Patients With Advanced Melanoma

For patients with advanced melanoma whose cancer has stopped responding to standard immunotherapy, treatment options have long been limited. A newly approved therapy by the U.S. Food and Drug Administration is changing that, offering new hope to patients with few remaining options. 

The FDA recently granted accelerated approval to RP1 in combination with nivolumab for adults with advanced melanoma whose disease has progressed after anti-PD-1 immunotherapy. The approval follows years of research, clinical trials and advocacy from physicians, patients and melanoma organizations, including Moffitt Cancer Center. 

Moffitt played an important role in the approval process. Nikhil Khushalani, MD, vice chair of Moffitt's Cutaneous Oncology Department, served as an adviser to drug manufacturer, Replimune, during the FDA review and participated in the agency's advisory committee meeting, where members voted 10-3 in favor of accelerated approval. The decision came after two previous FDA denials and months of advocacy from the melanoma community, including physicians, patient advocates and elected officials. 

Today, FDA granted approval to a genetically modified oncolytic viral therapy for adults with resistant advanced melanoma. This approval underscores FDA’s commitment to using every available regulatory tool to help bring meaningful treatment options to patients with serious… pic.twitter.com/XkYUTb0jHN

— U.S. FDA (@US_FDA) August 6, 2026

Filling a Critical Gap 

Immunotherapy has transformed melanoma care over the past decade, helping many patients live longer. But it doesn't work for everyone, and many patients who initially respond eventually develop resistance. 

Nikhil Khushalani, MD

"It is with great excitement that we now have a new treatment option for our patients with advanced melanoma whose disease has progressed following prior standard immunotherapy," Khushalani said. "While these drugs are excellent, unfortunately not all patients respond to them, and many eventually develop resistance and are in dire need of new treatment options." 

The newly approved treatment combines nivolumab with RP1, an engineered virus designed to infect and destroy melanoma cells while helping the immune system recognize and attack cancer throughout the body. The drug RP1 is injected directly into the tumor(s) while nivolumab is given intravenously.  

Clinical trial results showed meaningful and durable responses in patients whose melanoma had already progressed after standard immunotherapy, leading the FDA to grant accelerated approval while a larger confirmatory phase 3 study continues. Moffitt is one of the participating sites in that ongoing trial. 

Ready To Bring the Therapy to Patients 

Moffitt has spent the past year preparing for the approval and developing a streamlined process to offer the treatment as soon as it becomes commercially available. 

"We have a team here that has already defined a workflow as to how we are going to utilize this," Khushalani said. "We certainly hope to get this treatment to the appropriate patients as soon as it becomes commercially available." 

The center also plans to use image-guided procedures to deliver the therapy into tumors located deeper in the body, allowing more patients to benefit from the treatment. 

For Khushalani, the approval represents more than a scientific milestone. He says the FDA meeting underscored the importance of hearing directly from patients and families whose lives could be changed by new treatment options. 

"It was a concerted effort by the melanoma community, by patients, patient advocates, caregivers, physicians and elected officials because we felt this treatment was important to get to our patients," Khushalani said. "Hearing every story was incredibly moving. We are dealing with people facing a very difficult disease and if we are able to help get a new drug to them, then we've done something meaningful." 

While the approval is an important step forward, Khushalani says the work continues. 

"This is an important milestone, but our job is not done," he said. "We still need to find ways to help the patients who don't benefit from current treatments, and that's why we continue to invest in clinical trials and research."