Treatment Watch

treatment of adult and pediatric patients 2 years of age and older with hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA)

treatment of anemia in adults with alpha- or beta-thalassemia

treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms

treatment of pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available

Voyxact (sibeprenlimab) is a first-in-class APRIL inhibitor approved for reduction of proteinuria in adults with primary IgA nephropathy at risk for disease progression. It achieved a 51% placebo-adjusted reduction in proteinuria in the Phase 3 VISIONARY trial.

in combination with lenalidomide and rituximab for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL)

treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation who have no satisfactory alternative treatment options

in combination with atezolizumab or atezolizumab and hyaluronidase-tqjs, for the maintenance treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC) whose disease has not progressed after first-line induction therapy with atezolizumab or atezolizumab and hyaluronidase-tqjs, carboplatin and etoposide

treatment of adult patients with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment

treatment of pediatric patients 1 year of age and older with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in combination with chemotherapy

routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia B (congenital factor IX deficiency) with or without FIX inhibitors

treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU)

treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody

treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC)

replacement therapy in pediatric patients 5 years of age and older with adrenocortical insufficiency

treatment of generalized myasthenia gravis (gMG) in adult and pediatric patients 12 years of age and older who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive

treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB)

Vanrafia (atrasentan) is the first and only selective endothelin A receptor antagonist approved for reduction of proteinuria in adults with primary IgA nephropathy at risk of rapid disease progression. FDA accelerated approval based on Phase 3 ALIGN trial.

routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 years and older with hemophilia A or B with or without factor VIII or IX inhibitors

treatment of hyperphagia in adults and pediatric patients 4 years of age and older with Prader-Willi syndrome (PWS)

treatment of adults with complement 3 glomerulopathy (C3G), to reduce proteinuria

treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits

treatment of adults with idiopathic macular telangiectasia type 2 (MacTel)

Adult and pediatric patients aged 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas not amenable to complete resection. Approved based on the ReNeu Phase 2b trial (JCO 2025;43(6):716-729): ORR 41% adults, 52% pediatric. Significant pain reduction and QoL improvement. First MEK inhibitor approved for both adult and pediatric NF1-PN.