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.
Closing cancer health equity gaps require medical breakthroughs made possible by new funding approaches.
After a slow start, the race is on to inhibit key proteins and pathways within the complement system -- a biological cascade that is central to the innate immune system and many different diseases. As an added incentive, the one drug approved in this space is priced at $440,000 per year.