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.
Grafapex
TREOSULFAN
Medexus Pharma, Inc.
1 INDICATIONS AND USAGE GRAFAPEX is an alkylating drug indicated for: Use in combination with fludarabine as a preparative regimen for allogeneic hema…
Tyvaso
treprostinil
United Therapeutics
1 INDICATIONS AND USAGE Tyvaso is a prostacyclin mimetic indicated for the treatment of: Pulmonary arterial hypertension (PAH; WHO Group 1) to improve…
Tyvaso DPI
treprostinil inhalation powder
United Therapeutics Corporation
1 INDICATIONS AND USAGE Tyvaso DPI is a prostacyclin mimetic indicated for the treatment of: Pulmonary arterial hypertension (PAH; WHO Group 1) to imp…
Vesanoid
Tretinoin
Hoffmann-La Roche, Inc.
1 INDICATIONS AND USAGE Tretinoin capsules are indicated for the induction of remission in adults and pediatric patients 1 year of age and older with …
Triamcinolone Acetonide
TRIAMCINOLONE ACETONIDE
Alembic Pharmaceuticals Inc.
INDICATIONS AND USAGE Intramuscular Where oral therapy is not feasible, injectable corticosteroid therapy, including triamcinolone acetonide injectabl…
Egaten
triclabendazole
Novartis Pharmaceuticals Corporation
1 INDICATIONS AND USAGE EGATEN ® is indicated for the treatment of fascioliasis in patients 6 years of age and older. EGATEN ® tablet is an anthelmint…
Syprine
Trientine HCl
Merck Sharp & Dohme Research
INDICATIONS AND USAGE Syprine is indicated in the treatment of patients with Wilson’s disease who are intolerant of penicillamine. Clinical experience…
Cuvrior
trientine tetrahydrochloride
Orphalan
1 INDICATIONS AND USAGE CUVRIOR is indicated for the treatment of adult patients with stable Wilson's disease who are de-coppered and tolerant to peni…
LONSURF
trifluridine/tipiracil
Taiho Oncology, Inc.
1 INDICATIONS AND USAGE LONSURF is a combination of trifluridine, a nucleoside metabolic inhibitor, and tipiracil, a thymidine phosphorylase inhibitor…
Dojolvi
triheptanoin
Ultragenyx Pharmaceutical, Inc.
1 INDICATIONS AND USAGE DOJOLVI is indicated as a source of calories and fatty acids for the treatment of adult and pediatric patients with molecularl…
Neutrexin
Trimetrexate glucuronate
Medimmune Oncology, Inc.
Concurrent leucovorin administration as an alternative therapy for the treatment of moderate to severe PCP in immunocompromised patients who are intol…
Triptodur
triptorelin pamoate
Arbor Pharmaceuticals, LLC
1 INDICATIONS AND USAGE TRIPTODUR is indicated for the treatment of pediatric patients 2 years of age and older with central precocious puberty (CPP).…
Daybue
TROFINETIDE
Acadia Pharmaceuticals Inc.
1 INDICATIONS AND USAGE DAYBUE and DAYBUE STIX are indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and ol…
MembraneBlue
Trypan blue
Dutch Ophthalmic Research Center International BV
MembraneBlue 0.15% - Indications & Usage Section MembraneBlue™ 0.15% is indicated for use as an aid in ophthalmic surgery by staining the epiretinal m…
Tukysa
tucatinib
Seagen Inc.
1 INDICATIONS AND USAGE TUKYSA is a kinase inhibitor indicated: • in combination with trastuzumab and capecitabine for treatment of adult patients wit…
Ukoniq
umbralisib
TG Therapeutics, Inc.
treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based regimen (Ind…
Rinvoq
upadacitinib
AbbVie, Inc.
1 INDICATIONS AND USAGE RINVOQ/RINVOQ LQ is a Janus kinase (JAK) inhibitor. RINVOQ is indicated for the treatment of adults with moderately to severel…
Urea Cream 40 Percent
UREA
Method Pharmaceuticals, LLC
Indications and Usage For debridement and promotion of normal healing of hyperkeratotic surface lesions, particularly where healing is retarded by loc…
Uracin 20%
UREA 20%
PureTek Corporation
INDICATIONS AND USAGE For debridement and promotion of normal healing of hyperkeratotic surface lesions, particularly where healing is retarded by loc…
Urea 41
UREA 41
Oncora Pharma, LLC
INDICATIONS AND USAGE Urea 41% Cream is indicated for debridement and promotion of normal healing of hyperkeratotic surface lesions, particularly wher…
Dermacure
UREA 41%
Oncora Pharma, LLC
INDICATIONS & USAGE For debridement and promotion of normal healing of hyperkeratotic surface lesions, particularly where healing is retarded by local…
Urea 39.5% With 2% Salicylic Acid
UREA, SALICYLIC ACID
PureTek Corporation
INDICATIONS & USAGE For debridement and promotion of normal healing of hyperkeratotic surface lesions, particularly where healing is retarded by local…
URSO 250
Ursodiol
Aptalis Pharma US, Inc.
1 INDICATIONS AND USAGE URSO 250 and URSO Forte (ursodiol) tablets are indicated for the treatment of patients with primary biliary cholangitis (PBC).…
CNJ-016
Vaccinia Immune Globulin (Human) Intravenous
Cangene Corporation
1 INDICATIONS AND USAGE VIGIV (vaccinia immune globulin intravenous, human) is indicated for the treatment and/or modification of the following condit…
Ingrezza
valbenazine
Neurocrine Biosciences Inc.
1 INDICATIONS AND USAGE INGREZZA and INGREZZA SPRINKLE are indicated for the treatment of adults with: - tardive dyskinesia [see Clinical Studies ( 14…
ROCTAVIAN
VALOCTOCOGENE ROXAPARVOVEC-RVOX
BioMarin Pharmaceutical Inc.
1 INDICATIONS AND USAGE ROCTAVIAN is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with severe hemophilia …