FDA Approves Pralsetinib for RET Fusion Positive Metastatic NSCLC

The Food and Drug Administration (FDA) approved pralsetinib (Gavreto) for the treatment of patients with metastatic, RET fusion-positive non-small cell lung cancer (NSCLC), according to Genentech, the co-manufacturer of the drug.

“The FDA approval of Gavreto for RET fusion-positive non-small cell lung cancer is an important step towards our goal of providing an effective treatment option for every person diagnosed with lung cancer, no matter how rare or hard-to-treat their type of disease,” said Levi Garraway, MD, PhD, Genentech’s chief medical officer and head of Global Product Development, in a statement.

The accelerated approval was based off findings from the open-label, first-in-human phase 1/2 ARROW study, that showed that pralsetinib had durable responses in patients with rearranged during transfection (RET) fusion-positive NSCLC, regardless of whether or not they had previous therapy, RET fusion partner or central nervous system involvement.

The study had a 57% overall response rate. In the 87 people who were previously treated platinum-based chemotherapy, 5.7% of patients had a complete response, meaning that they had no detectable traces of cancer. In the 27 study participants who did not undergo previous treatment, 70% responded, and 11% had a complete response.

The most common adverse events (AEs) were fatigue, constipation, musculoskeletal pain, and increased blood pressure. More serious AEs included pneumonitis, hypertension, hepatotoxicity, hemorrhagic events, impaired wound healing, and fetal harm for women who are pregnant.

About 1-2% of patients with NSCLC have RET-activating fusions, which could drive cancers in the lungs and medullary thyroid. Currently, there are few treatment options for this patient population, so it is important that patients receive testing with the FDA-approved test to see if they are eligible for treatment with pralsetinib.

“We remain committed to finding personalized treatment options for people with cancer based on specific genomic or molecular alterations, and we look forward to partnering with Blueprint Medicines to further explore the potential of Gavreto across multiple RET-altered tumor types,” Garraway said.