Treatment Watch

eschar removal in adults with deep partial thickness (DPT) and/or full thickness (FT) thermal burns

treatment of adult patients with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy

treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test

prevention of recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older following antibiotic treatment for recurrent CDI

Treatment of adults with Hemophilia B (congenital Factor IX deficiency) who: currently use Factor IX prophylaxis therapy, or; have current or historical life-threatening hemorrhage, or; have repeated, serious spontaneous bleeding episodes

to delay the onset of Stage 3 type 1 diabetes in adults and pediatric patients 8 years of age and older with Stage 2 type 1 diabetes

treatment of neonatal seizures in term and preterm infants

treatment of adult patients with folate receptor-alpha (FR?) positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have received one to three prior systemic treatment regimens. Select patients for therapy based on an FDA-approved test.

as a single agent for the treatment of adult patients with histiocytic neoplasms

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

In combination with durvalumab for the treatment of adult patients with unresectable hepatocellular carcinoma (uHCC)

In combination with tremelimumab-actl for the treatment of adult patients with unresectable hepatocellular carcinoma (uHCC)

Treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements

Treatment of amyotrophic lateral sclerosis (ALS) in adults

To reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1 month of age and older with localized, non-metastatic solid tumors

To slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD)

To improve kidney function in adults with hepatorenal syndrome with rapid reduction in kidney function

Treatment of adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with fibroblast growth factor receptor 1 (FGFR1) rearrangement

Treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy

Routine prophylaxis to reduce the frequency of bleeding episodes in adults and children with hemophilia B (congenital Factor IX deficiency)

Treatment of seizures associated with Dravet syndrome (DS) in patients taking clobazam who are 6 months of age and older and weighing 7 kg or more

Treatment of chronic gout in adult patients refractory to conventional therapy

Treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older

Treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy

In combination with azacitidine or as monotherapy for the treatment of newly diagnosed acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy

Treatment of pulmonary arterial hypertension (PAH; WHO Group 1) to improve exercise ability

Treatment of pediatric patients aged one month and older with newly diagnosed juvenile myelomonocytic leukemia (JMML)

Treatment of adult and pediatric patients aged 12 years and older, weighing at least 40 kg, with eosinophilic esophagitis (EoE)

Treatment of human smallpox disease caused by variola virus in adults and pediatric patients weighing at least 3 kg

Treatment of adult patients with stable Wilson®s disease who are de-coppered and tolerant to penicillamine

Treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms

Treatment of seizures associated with Lennox-Gastaut syndrome (LGS) in patients 2 years of age and older

treatment of facial angiofibroma associated with tuberous sclerosis in adults and pediatric patients 6 years of age and older

Treatment of adult and pediatric patients 12 years of age or older with unresectable or metastatic melanoma

Treatment of adult patients with relapsed or refractory multiple myeloma, after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody

To decrease the need for red blood cell (RBC) transfusion due to hemolysis in adults with cold agglutinin disease (CAD)

For use in adults (age 18 and older) diagnosed with von Willebrand disease (VWD) for routine prophylaxis to reduce the frequency of bleeding episodes in patients with severe Type 3 VWD receiving on-demand therapy

treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma