FDA Approves First Cellular Therapy for Skin Cancer Patients

FDA Approves the First Cellular Therapy to Treat Patients with Unresectable or Metastatic Melanoma

Amtagvi, the first cellular therapy indicated for the treatment of adult patients with a type of skin cancer (melanoma) that is unable to be removed with surgery (unresectable) or has spread to other parts of the body (metastatic) and has previously been treated with other therapies, was approved today by the United States Food and Drug Administration.

“Unresectable or metastatic melanoma is an aggressive form of cancer that can be fatal,” stated Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “The approval of Amtagvi represents the culmination of scientific and clinical research efforts leading to a novel T cell immunotherapy for patients with limited treatment options.”

Melanoma is a type of skin cancer that is frequently induced by exposure to UV light, which can occur from either sunshine or indoor tanning. Although melanomas account for only around 1% of all skin malignancies, they cause a large number of cancer-related deaths. Melanoma can spread to other parts of the body if not diagnosed and treated promptly, resulting in metastatic illness.

Unresectable or metastatic melanoma may be treated with immunotherapy utilizing PD-1 inhibitors, which are antibodies that target certain proteins in the body to aid the immune system in fighting cancer cells. Furthermore, medications that target the BRAF gene, which aids in the regulation of cell development and function, may be utilized to treat melanoma caused by BRAF mutations. Patients whose melanoma has advanced despite these treatments have a significant unmet medical need.

Amtagvi is a tumor-derived autologous T cell immunotherapy made out of a patient’s own T cells, which help the immune system fight cancer. Prior to treatment, a surgical operation removes a piece of the patient’s tumor tissue. The patients’ T cells are extracted from the tumor tissue, produced, and then returned to the patient as a single dosage for infusion. This is the first time the FDA has approved tumor-derived T cell immunotherapy.

“Melanoma is a life-threatening cancer that can cause devastating impacts to affected individuals, with a significant risk of metastasizing and spreading to other areas in the body,” stated Nicole Verdun, M.D., director of the Office of Therapeutic Products at CBER. “Today’s approval reflects the FDA’s dedication and commitment to the development of innovative, safe, and effective treatment options for cancer patients.”

Amtagvi was approved under the Accelerated Approval pathway, which allows the FDA to approve drugs for serious or life-threatening illnesses or conditions if there is an unmet medical need and the drug is shown to have an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients (e.g., improving how patients feel or function, or whether they live longer). This method often allows patients to gain early access to a potential medicine while the company conducts additional trials to confirm the projected clinical benefit. A confirmatory trial is now underway to assess Amtagvi’s clinical benefit.

The safety and efficacy of Amtagvi were assessed in a global, multicenter, multicohort clinical trial involving adult patients with unresectable or metastatic melanoma who had previously received at least one systemic therapy, including a PD-1 blocking antibody, and, if positive for the BRAF V600 mutation, a BRAF inhibitor or a BRAF inhibitor combined with a MEK inhibitor. Effectiveness was determined using the objective response rate to treatment and the duration of response (measured from the date of confirmed initial objective response to the date of progression, death from any cause, beginning a new anti-cancer treatment, or discontinuation from follow-up, whichever occurred first). The objective response rate among the 73 patients treated with Amtagvi at the indicated dose was 31.5%, with three (4.1%) patients achieving a complete response and 20 (27.4%) obtaining a partial response. At six, nine, and twelve months, 56.5%, 47.8%, and 43.5% of patients who responded to treatment maintained their responses without tumor progression or death.

Patients on Amtagvi may experience sustained severe low blood count, severe infection, a heart issue, decreased respiratory or renal function, or fatal treatment-related complications. The label includes a boxed warning with information about these hazards. Patients taking this medicine should be thoroughly watched prior to and following the infusion for signs and symptoms of adverse reactions. In the presence of these symptoms, treatment should be withheld or discontinued as appropriate.

The most common side effects of Amtagvi were chills, fever, fatigue, tachycardia (abnormally fast heart rate), diarrhea, febrile neutropenia (fever associated with a low level of certain white blood cells), edema (swelling caused by fluid buildup in body tissues), rash, hypotension, hair loss, infection, hypoxia (abnormally low oxygen levels in the body), and feeling short of breath.

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