Veltuzumab

Roche’s Gazyva just increased its foothold in the autoimmune market with a fresh FDA approval , becoming the first new treatment option in 70 years for patients with idiopathic nephrotic syndrome (INS). The regulator cleared the anti-CD20 monoclonal antibody to reduce the risk of relapse in adults and children 2 and older with frequently relapsing or steroid-dependent, childhood-onset INS who are in complete remission. The FDA green light, Gazyva’s second autoimmune indication in less than 12 months, cementing the 2013 cancer drug’s second act in immune-mediated diseases. INS is a rare, kidney-related autoimmune disease marked by frequent relapses that can cause fatigue, swelling, weight gain and increased susceptibility to infections. It’s frequently diagnosed in childhood, and the relapses can drive anxiety, depression and reduced self-esteem in young patients who live in fear of unpredictable flare-ups. “For too long, patients living with INS have had to endure unpredictable relapses, prolonged steroid use, and the constant threat of disease progression,” said Andi Callaway, founder and president of The Nephrotic Syndrome Foundation. “This approval of Gazyva marks a shift that enables patients and families facing this debilitating disease to raise expectations for care and look ahead with optimism.” The FDA based its decision on data from Roche’s phase 3 INShore trial. In the trial, 95% of patients on Gazyva experienced no relapses after week 8 and were in complete remission at week 52, compared with 73% of patients on mycophenolate mofetil (MMF). The drug also succeeded on secondary endpoints, with statistically significant and clinically meaningful improvements in overall relapse-free survival (RFS) and median time to first relapse or death, as well as fewer relapses from baseline to week 52, compared with MMF. “By directly targeting B cells to more effectively control disease activity, this first-of-its-kind therapy equips healthcare providers with an option that provides the possibility of lasting remission, giving families the freedom to focus on their child’s growth and potential,” said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of global product development. INS is Gazyva’s second immune-mediated indication, following an approval last October in adult lupus nephritis, a potentially fatal kidney inflammation caused by systemic lupus erythematosus (SLE), the most common form of lupus. The ongoing autoimmune expansion represents a second act for the older drug, which was originally approved in 2013 for a narrow leukemia indication. The new approval is unlikely to be the end of Roche’s reinvention plans for the drug. Gazyva is currently under FDA review for two more autoimmune conditions: SLE and primary membranous nephropathy (pMN), a kidney disease for which there is currently no FDA-approved therapy. The SLE application followed a positive phase 3 readout announced last November, while Roche reported a phase 3 win in pMN in February. Roche is also investigating the drug in pediatric lupus nephritis. If the autoimmune pivot continues to be successful, GlobalData has estimated Gazyva sales could reach $1.8 billion or more by 2030.