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.
Tzield
teplizumab-mzwv
Provention Bio, Inc.
1 INDICATIONS AND USAGE TZIELD is indicated to delay the onset of Stage 3 type 1 diabetes in adults and pediatric patients 8 years of age and older wi…
Tepmetko
tepotinib
EMD Serono, Inc.
1 INDICATIONS AND USAGE TEPMETKO is indicated for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) harboring mesench…
Parathar
Teriparatide
Rhone-Poulenc Rorer Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE Teriparatide injection is indicated: For the treatment of postmenopausal women with osteoporosis at high risk for fracture (de…
Terlivaz
terlipressin
Mallinckrodt Pharmaceuticals Ireland Ltd
1 INDICATIONS AND USAGE TERLIVAZ is indicated to improve kidney function in adults with hepatorenal syndrome with rapid reduction in kidney function. …
Xenazine
tetrabenazine
Lundbeck
1 INDICATIONS AND USAGE XENAZINE is indicated for the treatment of chorea associated with Huntington’s disease. XENAZINE is a vesicular monoamine tran…
SYMDEKO
TEZACAFTOR AND IVACAFTOR
Vertex Pharmaceuticals Incorporated
1 INDICATIONS AND USAGE SYMDEKO is indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who are homozygous for the F…
Thalomid
Thalidomide
Celgene Corporation
1 INDICATIONS AND USAGE • THALOMID in combination with dexamethasone is indicated for the treatment of patients with newly diagnosed multiple myeloma …
TEPADINA
THIOTEPA
Amneal Pharmaceuticals LLC
1 INDICATIONS AND USAGE TEPADINA (thiotepa) is an alkylating drug indicated: To reduce the risk of graft rejection when used in conjunction with high-…
Thyrogen
Thyrotropin alfa
Genzyme Corporation
1 INDICATIONS AND USAGE THYROGEN ® is a thyroid stimulating hormone indicated for: Adjunctive Diagnostic Tool for Well-Differentiated Thyroid Cancer: …
Tindamax
Tinidazole
Presutti Laboratories, Inc.
1 INDICATIONS AND USAGE 1 INDICATIONS AND USAGE 1.1 Trichomoniasis Tinidazole is indicated for the treatment of trichomoniasis caused by Trichomonas v…
Thiola
Tiopronin
Mission Pharmacal Company
1 INDICATIONS AND USAGE THIOLA EC is indicated, in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine st…
Kymriah
tisagenlecleucel
Novartis Pharmaceuticals Corporation
1 INDICATIONS AND USAGE KYMRIAH is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of: Patients up to…
Tevimbra
tislelizumab-jsgr
BeOne Medicines USA, Inc.
1 INDICATIONS AND USAGE TEVIMBRA is a programmed death receptor-1 (PD-1)-blocking antibody indicated for: Esophageal Cancer in combination with platin…
TOBRAMYCIN
TOBRAMYCIN
Aurobindo Pharma Limited
1 INDICATIONS AND USAGE Tobramycin inhalation solution is indicated for the management of cystic fibrosis in adults and pediatric patients 6 years of …
TOBRADEX ST
TOBRAMYCIN / DEXAMETHASONE
Harrow Eye, LLC
1 INDICATIONS AND USAGE TOBRADEX ST ophthalmic suspension is indicated for steroid-responsive inflammatory ocular conditions for which a corticosteroi…
TOBI
Tobramycin for inhalation
Mylan Specialty LP
1 INDICATIONS AND USAGE TOBI is indicated for the management of cystic fibrosis in adults and pediatric patients 6 years of age and older with Pseudom…
Actemra
tocilizumab
Genentech, Inc.
1 INDICATIONS AND USAGE ACTEMRA ® (tocilizumab) is an interleukin-6 (IL-6) receptor antagonist indicated for treatment of: Rheumatoid Arthritis (RA) (…
Qalsody
tofersen
Biogen Inc.
1 INDICATIONS AND USAGE QALSODY is indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide …
Jynarque
tolvaptan
Otsuka Pharmaceutical Company, Ltd.
1 INDICATIONS AND USAGE JYNARQUE is indicated to slow kidney function decline in adults at risk of rapidly progressing autosomal dominant polycystic k…
Topamax
Topiramate
Johnson & Johnson Pharmaceutical R & D, LLC
1 INDICATIONS AND USAGE TOPAMAX is indicated for: Epilepsy: initial monotherapy for the treatment of partial-onset or primary generalized tonic-clonic…
Fareston
Toremifene
Orion Corporation
1 INDICATIONS AND USAGE FARESTON® is an estrogen agonist/antagonist indicated for the treatment of metastatic breast cancer in postmenopausal women wi…
Loqtorzi
toripalimab-tpzi
Coherus BioSciences, Inc.
1 INDICATIONS AND USAGE LOQTORZI is a programmed death receptor-1 (PD-1)- blocking antibody indicated: in combination with cisplatin and gemcitabine, …
Bexxar
Tositumomab and iodine I 131 tositumomab
GlaxoSmithKline LLC
Treatment of patients with CD20 positive, follicular, non-Hodgkin's lymphoma, with and without transformation, whose disease is refractory to Rituxima…
Ojemda
tovorafenib
Day One Biopharmaceuticals, Inc.
1 INDICATIONS AND USAGE OJEMDA is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade gl…
Yondelis
trabectedin
Janssen Research & Development, LLC
1 INDICATIONS AND USAGE YONDELIS ® is indicated for the treatment of adult patients with unresectable or metastatic liposarcoma or leiomyosarcoma who …
MEKINIST
trametinib
Novartis Pharmaceuticals Corp.
1 INDICATIONS AND USAGE MEKINIST is a kinase inhibitor indicated as a single agent for the treatment of BRAF-inhibitor treatment-naïve patients with u…
MEKINIST and TAFINLAR
trametinib and dabrafenib
Novartis Pharmaceuticals Corporation
TAFINLAR is indicated, in combination with trametinib, for the adjuvant treatment of patients with melanoma with BRAF V600E or V600K mutations, as det…
tranexamic acid
TRANEXAMIC ACID
Avenacy, Inc.
1 INDICATIONS AND USAGE Tranexamic acid injection, USP is indicated in patients with hemophilia for short-term use (2 to 8 days) to reduce or prevent …
Herceptin
trastuzumab
Genentech, Inc.
1 INDICATIONS AND USAGE Herceptin is a HER2/neu receptor antagonist indicated in adults for: The treatment of HER2-overexpressing breast cancer. ( 1.1…
Imjudo
tremelimumab-actl
AstraZeneca Pharmaceuticals LP
1 INDICATIONS AND USAGE IMJUDO is a cytotoxic T-lymphocyte-associated antigen 4 (CTLA-4) blocking antibody indicated: • in combination with durvalumab…