Last month, Novartis (NVS -3.20%) revealed encouraging results from a phase 3 clinical trial of the rare-disease drug candidate known as iptacopan for adult patients diagnosed with a blood disorder known as paroxysmal nocturnal hemoglobinuria (PNH).
What could these results mean for PNH patients and Novartis’ future? Let’s dig into the data from the clinical trial and the global PNH market to get an answer.
A potentially life-changing treatment
PNH is a rare and serious blood disorder that’s estimated to impact 10 to 20 people per million around the world. The disease causes red blood cells (RBCs) to break apart, which can lead to symptoms such as muscle spasms, fatigue, shortness of breath, and unintentional weight loss.
As with any medical condition, the severity of PNH can vary greatly. Worst-case scenarios can include potentially fatal blood clots. This is why it’s of utmost…
