Plozasiran, a first-in-class RNA interference therapeutic, reduced triglycerides by 80% and lowered the risk of acute pancreatitis by 83% in patients with familial chylomicronemia syndrome, earning FDA...
Crinecerfont, a first-in-class therapy, improved androgen control and reduced reliance on high-dose glucocorticoids for patients with congenital adrenal hyperplasia.
The once-monthly RNA interference injection medication received FDA approval based on results from the phase II PHYOX2 clinical trial and phase III PHYOX3 extension study.
The US Food and Drug Administration has approved the drug based on the results from the KEEPsAKE-1 and KEEPsAKE-2 randomized, placebo-controlled studies.
A new therapeutic agent added to standard of care significantly improves outcomes for patients with serious complication of systemic lupus erythematosus.
