FDA Approves First Cell Therapy to Treat Melanoma

The U.S. Food and Drug Administration (FDA) has approved Iovance Biotherapeutics’ Amtagvi, the first cellular therapy indicated for the treatment of adult patients with melanoma that is unable to be removed with surgery (unresectable) or has spread to other parts of the body (metastatic) that previously has been treated with other therapies (a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor). 

Approval was based on a global, multicenter, multicohort clinical study including adult patients with unresectable or metastatic melanoma that evaluated whether Amtagvi established an objective response rate to treatment and duration of response (measured from the date of confirmed initial objective response to the date of progression, death from any cause, starting a new anti-cancer treatment or discontinuation from follow-up, whichever came first). Among the 73 patients treated with Amtagvi at the recommended dose, the objective response rate was 31.5 percent, including three (4.1 percent) patients with a complete response and 20 (27.4 percent) patients with a partial response. Among patients who were responsive to the treatment, 56.5 percent, 47.8 percent and 43.5 percent continued to maintain responses without tumor progression or death at six, nine and 12 months, respectively.

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

According to FDA, patients treated with Amtagvi may exhibit prolonged severe low blood count, severe infection, cardiac disorder or develop worsened respiratory or renal function or have fatal treatment-related complications. A boxed warning is included in the label containing information about these risks. Patients receiving this product should be closely monitored before and after infusion for signs and symptoms of adverse reactions. Treatment should be withheld or discontinued in the presence of these symptoms, as indicated.

Amtagvi was approved through the accelerated approval pathway, under which FDA may approve drugs for serious or life-threatening illnesses or conditions where 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 (improving how patients feel or function, or whether they survive longer).