The Food and Drug Administration (FDA) has granted traditional approval to Retevmo (selpercatinib) for the treatment of adult and pediatric patients 2 years old and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory, if radioactive iodine is appropriate, the agency has announced.
Retevmo had previously been granted accelerated approval for patients at least 12 years old with this indication in 2020.
The efficacy of Retevmo, the agency reported, was evaluated in the LIBRETTO-001 clinical trial, which showed an overall response rate (ORR; patients who responded partially or completely to treatment) of 85% among 41 pretreated patients and 96% among 24 patients who had not previously received systemic therapy. The median durations of response were 26.7 months in the pretreated group. In the previously untreated group, the median duration of response was not estimable, meaning more than half of systemic therapy-naïve patients were still responding to treatment at the time of data collection.
Evidence, the agency noted, also included data from 10 pediatric and young adult patients in the LIBRETTO-121 clinical trial, which had an ORR of 60%, with 83% having a response of at least a year.
Side effects occurring in at least a quarter of patients included edema, diarrhea, fatigue, dry mouth, hypertension, abdominal pain, constipation, rash, nausea and headache. The most common grade 3 (severe) or 4 (life-threatening) laboratory abnormalities were decreased lymphocytes, increased alanine aminotransferase (ALT), increased aspartate aminotransferase (AST), decreased sodium and decreased calcium.
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