Shots:
The US FDA approved Myqorzo (aficamten) is now commercially available in the US for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms
Myqorzo is a QD, oral cardiac myosin inhibitor available in 5, 10, 15, and 20 mg tablets and directly targets hypercontractility and LVOT obstruction…
Shots:
The US FDA has approved Agios’ Aqvesme (mitapivat) for the treatment of anemia in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia
Approval was based on the global P-III (ENERGIZE) and (ENERGIZE-T) studies (n=452), which met all 1EPs and 2EPs, demonstrating significant improvements in hemoglobin, fatigue, and reductions in transfusion burden vs PBO
Aqvesme…
Shots:
The US FDA has granted accelerated approval to Vanrafia for proteinuria reduction in adults with primary IgAN at risk of rapid disease progression based on the ongoing P-III (ALIGN) trial, with no requirement for REMS program; traditional approval will be supported by eGFR results expected in 2026
Trial assessed atrasentan (0.75mg, PO, QD) +…

