Treatment Watch

PROCYSBI is indicated for the treatment of nephropathic cystinosis in adults and pediatric patients 1 year of age and older.

To reduce the frequency of painful crises and to reduce the need for blood transfusions in pediatric patients, 2 years of age and older, with sickle cell anemia with recurrent moderate to severe painful crises

For the diagnosis of adult growth hormone deficiency

an adeno-associated virus vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Patients must have viable retinal cells determined by a treating physician

Routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors

ALECENSA is indicated for the treatment of patients with anaplastic lymphoma kinase (ALK)-positive, metastatic non-small cell lung cancer (NSCLC) as detected by an FDA-approved test.

Treatment of patients with Erdheim-Chester Disease with BRAF V600 mutation

for the treatment of adult patients with generalized Myasthenia Gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody positive

for the treatment of scurvy in adult and pediatric patients age 5 months and older for whom oral administrtaion is not possible, insufficient or contraindicated

DESCOVY, indicated in combination with other antiretroviral agents, for the treatment of HIV-1 infection in adults and pediatric patients weighing at least 35 kg and also indicated, in combination with other antiretroviral agents other than protease inhibitors that require a CYP3A inhibitor, for the treatment of HIV-1 infection in pediatric patients weighing at least 25 kg and less than 35 kg.

Treatment of adult patients with relapsed follicular lymphoma who have received at least two prior systemic therapies

Treatment of newly-diagnosed CD33-positive acute myeloid leukemia in adults and treatment of relapsed or refractory CD33-positive acute myeloid leukemia in adults and in pediatric patients 2 years and older

Treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse

treatment of dyskinesia in patients with Parkinson's disease receiving levodopa-based therapy with or without concomitant dopminergic medications

Maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer, who are in a complete or partial response to platinum-based chemotherapy.

Treatment of adult patients with relapsed or refractory acute myeloid leukemia with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test

To reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older.

Treatment of pediatric patients 2 years of age and older with central precocious puberty

Mekinist in combination with dabrafenib, for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with BRAF V600E mutation as detected by an FDA-approved test.

For routine prophylaxis to prevent Hereditary Angioedema (HAE) attacks in adolescent and adult patients.

Treatment of adult patients with previously untreated and previously treated CLL in combination with fludarabine and cyclophosphamide

Used in combination with other antiretroviral agents for the treatment of patients with HIV-1 infection

Optical imaging agent indicated in patients with glioma (suspected World Health Organization Grades III or IV on preoperative imaging) as an adjunct for the visualization of malignant tissue during surgery

Treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are anaplastic lymphoma kinase (ALK)-positive as detected by an FDA-approved test

Treatment of amyotrophic lateral sclerosis (ALS)

Steritalc is indicated in adults to decrease the recurrence of pneumothorax.

Treatment of patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib

Treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation-positive as detected by an FDA approved test, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation.

Treatment of patients with hepatocellular carcinoma (HCC or liver cancer) who have been previously treated with sorafenib.

Management of pediatric patients with active polyarticular juvenile idiopathic arthritis (pJIA) who have had an insufficient therapeutic response to, or are intolerant of, an adequate trial of first-line therapy including full dose non-steroidal anti-inflammatory agents (NSAIDs).

Treatment of chronic HCV genotype 2 or 3 infection in pediatric patients 12 years of age and older or weighing at least 35 kg without cirrhosis or with compensated cirrhosis for use in combination with ribavirin

indicated in adults for the treatment of tardive dyskinesia

Indicated for maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy

Treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma

Treatment of carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSA therapy.

Treatment of patients with Marginal Zone Lymphoma (MZL) who require systemic therapy and have received at least one prior anti-CD20-based therapy (indication withdrawn)