Novartis Scores Breakthrough Therapy Designation to Speed Prostate Cancer Drug Approval


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The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to an experimental prostate cancer treatment in Phase III development.

On Wednesday, Novartis announced the designation given to experimental drugs that show potential to substantially improve treatment options over available therapies. It was granted to Lu-PSMA-617, an investigational radioligand therapy to treat metastatic castration-resistant prostate cancer (mCRPC). The designation was based on positive data from the Phase III VISION study that combined Lu-PSMA-617 with standard-of-care in patients with progressive PSMA-positive mCRPC.

Novartis highlighted data from the Phase III VISION study at the American Society of Clinical Oncology meeting earlier this month. Data showed Lu-PSMA-617, a targeted radioligand therapy, plus best standard of care (SOC), demonstrated significant improvement in overall survival in patients with progressive PSMA-positive metastatic castration-resistant prostate cancer (mCRPC). 

Lu-PSMA-617 plus SOC was evaluated against SOC alone. Novartis said the OS difference between the Lu-PSMA-617 combination and SOC was statistically significant, with an estimated 38% reduction in risk of death in the 177Lu-PSMA-617 arm. 

Patients who received Lu-PSMA-617 also demonstrated a statistically significant 60% risk reduction for radiographic progression-free survival or death, compared to the best standard of care only arm. The five-year survival rate for patients with metastatic prostate cancer is about 30%. 

At ASCO, John Tsai, Head of Global Drug Development and Chief Medical Officer for Novartis, said the VISION study is the first to demonstrate the potential of a radioligand therapy in advanced prostate cancer. 

Lu-PSMA-617 combines a targeting ligand with a therapeutic radioisotope. Once the treatment is administered, Lu-PSMA-617 binds to prostate cancer cells that express PSMA, a transmembrane protein, with high tumor-to-normal tissue uptake. Once bound, emissions from the radioisotope damage tumor cells and disrupt the cell’s ability to replicate, leading to cell death. The radiation from the radioisotope operates over very short distances to limit damage to surrounding cells. 

Novartis is planning to assess Lu-PSMA-617 in earlier line treatment settings for metastatic prostate cancer. The company will investigate the radioligand therapy in the mCRPC pre-taxane setting (PSMAfore) and the metastatic hormone-sensitive setting (PSMAddition).

Earlier this month, the FDA awarded the Breakthrough Therapy designation to Protagonist Therapeutics and Janssen. Protagonist’s lead drug candidate rusfertide is being assessed as a potential treatment of patients with polycythemia vera (PV) for the reduction of erythrocytosis. Specifically, the drug candidate is being developed for those patients who no longer require additional treatment for thrombocytosis and/or leukocytosis. Previously, the FDA granted orphan drug status and Fast Track Designation to rusfertide in this indication.

Janssen, a division of Johnson & Johnson, received the designation for its multiple myeloma drug, teclistamab. Janssen’s teclistamab is an off-the-shelf, T-cell redirecting, a bispecific antibody that targets both B-cell maturation antigen (BCMA) and CD3 receptors. Teclistamab is currently being evaluated in a Phase II clinical study for the treatment of relapsed or refractory multiple myeloma and is also being explored in combination studies. 



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