The FDA approves a new therapy for difficult to treat metastatic melanoma

The FDA approves a new therapy for difficult-to-treat metastatic melanoma

Iovance Biotherapeutics,

The AMTAGVI (lifileucel) product packaging is shown in this handout photo from Iovance Biotherapeutics


On Friday, the U.S. Food and Drug Administration approved a new, first-of-its-kind therapy that could help patients with metastatic melanoma, a rare but deadly form of skin cancer.

The drug, made by California-based Iovance Biotherapeutics, is approved to treat patients in whom other medications have failed and the skin cancer cannot be removed with surgery or the cancer has spread to other parts of the body.

Amtagvi, as the treatment is called, is the first cell therapy to treat this form of solid tumor cancer. The company said the drug is manufactured in Philadelphia and could help up to “several thousand patients per year.”

The therapy uses a person's own immune cells to fight the cancer. In this treatment, doctors remove tissue from the patient's tumors, take immune cells from them and grow them in the laboratory. Once there are enough immune cells, doctors give them back to the patient in an infusion and the immune cells overwhelm and destroy the cancer.

Patients only need to receive the treatment once for it to work – and it can work for years, Dr. Ryan Sullivan, deputy director of the melanoma program at Mass General Cancer Center. The center was the site of one of the trials of the new therapy, which was evaluated in several centers around the world.

In the studies, the FDA said that “among the 73 patients treated with Amtagvi at the recommended dose, the objective response rate was 31.5%, including three (4.1%) patients with complete responses and 20 ( 27.4%) patients with partial response.” Answer. Of the patients who responded to treatment, 56.5%, 47.8% and 43.5% continued to respond without tumor progression or death at six, nine and 12 months, respectively.”

The FDA said the treatment will come with a warning label to inform patients that the treatment can cause serious bleeding, serious infections and cardiovascular problems.

Other risks associated with the treatment include the surgery associated with the treatment and the seven days of intensive chemotherapy that the patient needs before treatment.

The company states that the other side effects may include chills, fever, fatigue, fast heartbeat, diarrhea, fever, rash, and hair loss. Most side effects subside in the first few weeks. Despite the risks, doctors say the benefits for patients could be enormous.

“So patients not only have an extension of two or three weeks, but they can also be cured by the treatment or at least have the disease under control, and that will take another two, three, four years and longer,” said Sullivan, who is also an associate professor at Harvard Medical School. “That’s really exciting.”

Long-term follow-up studies will be needed to show how long treatment can last.

According to the American Cancer Society, approximately 100,640 new melanomas are diagnosed each year, and more than 8,000 people die from melanoma each year in the United States. This cancer accounts for only 1% of skin cancer cases, but is responsible for many of the skin cancer-related deaths.

“Melanoma is a life-threatening cancer that can have devastating effects on those affected and carries a significant risk of metastasis and spread to other areas of the body,” said Dr. Nicole Verdun, director of the Office of Therapeutic Products at the Center of Biologics Evaluation and Research. “Today’s approval reflects the FDA’s dedication and commitment to developing innovative, safe and effective treatments for cancer patients.”

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Research suggests that this approach to cancer treatment could also be useful in treating other difficult-to-fight cancers.

“Given the significant unmet need in the advanced melanoma community, we are proud to offer these patients a personalized, unique treatment option,” said Dr. Frederick Vogt, interim CEO and president of Iovance. “We are continuing our development efforts to address additional unmet medical needs in patients with solid tumor cancers and to bring our novel cell therapies to more patients with melanoma and other cancers.”

Sullivan, who treats melanoma patients in Massachusetts, said Friday's FDA announcement will be good news.

“While melanoma patients have many more treatment options than they did 15 years ago, many of our patients diagnosed with metastatic melanoma still die,” Sullivan said. “It's a very good day to have another option, especially an option for a patient population where our standard therapies have failed.”