Novartis’s prostate cancer drug 177Lu-PSMA-617 granted FDA breakthrough designation

Data from trial show the potential first-in-class radioligand therapy substantially enhanced overall survival.

This comes on the back of results from its pivotal, Phase III VISION study that evaluated the targeted radioligand therapy when used alongside standard of care (SOC) in comparison to SOC alone, in patients with progressive PSMA-positive metastatic castration-resistant prostate cancer (mCRPC). The FDA awards breakthrough designation for medicines being evaluated for serious conditions where early clinical evidence indicates the potential for substantial improvement over available therapy. The radioligand therapy (RLT) which targets the prostate-specific membrane antigen (PSMA), present in the majority of patients with mCRPC, had shown promising Phase II data previously - inspiring Novartis’s $2.1 billion acquisition for the drug’s then developer Endocyte back in 2018 as it looked to expand its RLT platform.

Designed to enrol 831 participants, the VISION trial is expected to conclude in June 2022. Participants are randomized 1:1 to receive either 177Lu-PSMA-617 intravenously six weekly for a maximum of six cycles or best supportive care or best SOC alone. Best supportive care or best SOC could also be used in the experimental arm. Results recently released from the Phase III trial show the drug impressed in relation to its two primary end points: overall survival (OS) and radiographic progression-free survival (rPFS). The median OS in the experimental arm was 15.3 months compared to 11.3 months in the SOC alone arm, and the rPFS for the experimental arm was also higher at 8.7 months compared to 3.4 months in the SOC-only arm.