FDA-Approved Treatments
Drugs with orphan designation or indications for rare diseases — 970 total
Drug information is sourced from FDA labels and prescribing information. Always verify current prescribing information with your pharmacist or prescriber before starting, stopping, or changing any medication.
Chemet capsules
Succimer
Bock Pharmacal Company
1 INDICATIONS AND USAGE CHEMET is indicated for the treatment of lead poisoning in pediatric patients aged 1 year and older with blood lead levels abo…
Enjaymo
SUTIMLIMAB-JOME
Recordati Rare Diseases, Inc.
1 INDICATIONS AND USAGE ENJAYMO is a classical complement inhibitor indicated for the treatment of hemolysis in adults with cold agglutinin disease (C…
Chirostim
Synthetic human secretin
ChiRhoClin, Inc.
Stimulation of pancreatic secretions, including bicarbonate, to aid in the diagnosis of pancreatic exocrine dysfunction, stimulation of gastrin secret…
Secreflo
Synthetic porcine secretin
ChiRhoClin, Inc.
Use in secretin stimulation testing for stimulation of gastrin secretion to aid in the diagnosis of gastrinoma ||| Use in secretin stimulation testing…
Envarsus XR
tacrolimus
Veloxis Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE ENVARSUS XR is a calcineurin-inhibitor immunosuppressant indicated for: The prophylaxis of organ rejection in de novo kidney t…
Astagraf XL
tacrolimus extended-release capsules
Astellas Pharma Global Development, Inc.
1 INDICATIONS AND USAGE ASTAGRAF XL ® is indicated for the prophylaxis of organ rejection in kidney transplant patients in combination with other immu…
Tadalafil
TADALAFIL
Northwind Health Company, LLC
1 INDICATIONS AND USAGE Tadalafil is a phosphodiesterase 5 (PDE5) inhibitor indicated for the treatment of pulmonary arterial hypertension (PAH) (WHO …
Vyndaqel
TAFAMIDIS MEGLUMINE
Pfizer Laboratories Div Pfizer Inc
1. INDICATIONS AND USAGE VYNDAQEL and VYNDAMAX are indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated…
MONJUVI
tafasitamab-cxix
Incyte Corporation
1 INDICATIONS AND USAGE MONJUVI is a CD19-directed cytolytic antibody indicated: in combination with lenalidomide for the treatment of adult patients …
Krintafel
tafenoquine
GlaxoSmithKline Intellectual Property Development Ltd. England
1 INDICATIONS AND USAGE KRINTAFEL is indicated for the radical cure (prevention of relapse) of Plasmodium vivax malaria in patients aged 16 years and …
ELZONRIS
Tagraxofusp-erzs
Stemline Therapeutics, Inc.
1 INDICATIONS AND USAGE ELZONRIS is indicated for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric pat…
Ibtrozi
taletrectinib
Nuvation Bio Inc.
1 INDICATIONS AND USAGE IBTROZI ™ (taletrectinib) is indicated for the treatment of adult patients with locally advanced or metastatic ROS1 -positive …
ELELYSO
TALIGLUCERASE ALFA
Pfizer Laboratories Div Pfizer Inc
1 INDICATIONS AND USAGE ELELYSO is indicated for the treatment of patients 4 years of age and older with a confirmed diagnosis of Type 1 Gaucher disea…
IMLYGIC
talimogene laherparepvec
BioVex, Inc. (subsidiary of Amgen)
1 INDICATIONS AND USAGE IMLYGIC is a genetically modified oncolytic viral therapy indicated for the local treatment of unresectable cutaneous, subcuta…
Talvey
talquetamab-tgvs
Janssen Biotech, inc.
1 INDICATIONS AND USAGE TALVEY is indicated for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at leas…
Tamoxifen Citrate
TAMOXIFEN CITRATE
A-S Medication Solutions
INDICATIONS AND USAGE Metastatic Breast Cancer: Tamoxifen citrate tablets, USP are effective in the treatment of metastatic breast cancer in women and…
Imdelltra
tarlatamab-dlle
Amgen Inc.
1 INDICATIONS AND USAGE IMDELLTRA is indicated for the treatment of adult patients with extensive stage small cell lung cancer (ES-SCLC) with disease …
HETLIOZ
tasimelteon
Vanda Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE HETLIOZ is a melatonin receptor agonist. HETLIOZ capsules are indicated for the treatment of: Non-24-Hour Sleep-Wake Disorder …
Tazverik
tazemetostat
Epizyme, Inc.
1 INDICATIONS AND USAGE TAZVERIK is a methyltransferase inhibitor indicated for the treatment of: Adults and pediatric patients aged 16 years and olde…
Kimmtrak
tebentafusp-tebn
Immunocore Ltd
1 INDICATIONS AND USAGE KIMMTRAK is indicated for the treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma.…
LYMPHOSEEK
technetium Tc 99m tilmanocept
Cardinal Health 414, LLC
1 INDICATIONS AND USAGE Lymphoseek is a radioactive diagnostic agent indicated with or without scintigraphic imaging for: • Lymphatic mapping using a …
Technetium Tc99m Sulfur Colloi
Technetium Tc99m sulfur colloid injection, lyophilized
Pharmalucence, Inc.
Localization of lymph nodes draining a primary tumor in patients with melanoma when used with a hand-held gamma counter…
Tecvayli
teclistamab-cqyv
Janssen Biotech, Inc.
1 INDICATIONS AND USAGE TECVAYLI is indicated for the treatment of adult patients with relapsed or refractory multiple myeloma in combination with dar…
TPOXX
tecovirimat
SIGA Technologies, Inc.
1 INDICATIONS AND USAGE TPOXX is an inhibitor of the orthopoxvirus VP37 envelope wrapping protein and is indicated for the treatment of human smallpox…
GATTEX
teduglutide [rDNA origin]
Takeda Pharmaceuticals U.S.A., Inc.
1 INDICATIONS AND USAGE GATTEX ® is indicated for the treatment of adults and pediatric patients 1 year of age and older with Short Bowel Syndrome (SB…
Xermelo
telotristat etiprate
TerSera Therapeutics, LLC
1 INDICATIONS AND USAGE Xermelo is indicated for the treatment of carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in…
Temodar
temozolomide
Schering-Plough Research Institute
1 INDICATIONS AND USAGE TEMODAR is an alkylating drug indicated for the treatment of adults with: Newly diagnosed glioblastoma concomitantly with radi…
Torisel
Temsirolimus
Wyeth Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE TORISEL is indicated for the treatment of advanced renal cell carcinoma. TORISEL ® is a kinase inhibitor indicated for the tre…
Vumon for injection
Teniposide
Bristol-Myers Squibb Pharmaceutical Research Institute
Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents.…
Viread
tenofovir
Gilead Sciences, Inc.
1 INDICATIONS AND USAGE VIREAD is a nucleotide analog HIV-1 reverse transcriptase inhibitor and an HBV reverse transcriptase inhibitor and is indicate…