FDA approves new treatments for dermatomyositis and polycythemia vera

The US Food and Drug Administration has approved two new treatments for rare conditions.

First, the FDA approved Lisraya (brepocitinib) for adults with dermatomyositis, a rare autoimmune disease affecting skin and muscles. As the first targeted therapy for this condition, Lisraya acts as a dual TYK2 and JAK1 inhibitor. Clinical trials indicated that the once-daily oral treatment improved disease activity and helped reduce patient dependence on corticosteroids.

Second, the FDA approved Mimrylo (rusfertide), developed by Takeda Pharmaceuticals, for adults with polycythemia vera. This first-in-class treatment mimics the hormone hepcidin to regulate iron availability, thereby limiting the overproduction of red blood cells. The therapy aims to reduce the need for frequent therapeutic phlebotomy, a common procedure used to manage the blood thickening associated with the disorder.