Novartis drug iptacopan, which won its first FDA approval in early December in a rare blood disorder, has met the main goal of a pivotal test in an ultra-rare kidney disease. The small molecule’s potential to address many diseases has stirred up blockbuster expectations.
FDA approval of Novartis’s Fabhalta makes the drug the first approved oral therapy for rare blood disorder paroxysmal nocturnal hemoglobinuria. With clinical data showing superiority versus two infused AstraZeneca drugs, Novartis’s pill is well-positioned to take market share from those blockbuster products.
Chee Kay Cheung, MBCHB, MRCP, PhD, discusses unmet needs in the management of IgAN, commenting on the need for new medication and highlighting budesonide and SGLT2 inhibitors.