FDA-Approved Treatments
Drugs with orphan designation or indications for rare diseases — 955 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.
Deferasirox oral
DEFERASIROX ORAL
Ascend Laboratories, LLC
1 INDICATIONS AND USAGE Deferasirox is an iron chelator indicated for the treatment of chronic iron overload due to blood transfusions in patients 2 y…
FERRIPROX
DEFERIPRONE
Chiesi USA, Inc.
1 INDICATIONS AND USAGE FERRIPROX Tablets are indicated for the treatment of transfusional iron overload in adult and pediatric patients 8 years of ag…
Defitelio
defibrotide
Jazz Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE DEFITELIO is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known…
Deflazacort Oral Suspension
DEFLAZACORT
Zydus Pharmaceuticals (USA) Inc.
1 INDICATIONS AND USAGE Deflazacort oral suspension is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and…
Deflazacort Oral Suspension
DEFLAZACORT ORAL
Sun Pharmaceutical Industries, Inc.
1 INDICATIONS AND USAGE Deflazacort oral suspension is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and…
Ablysinol
dehydrated alcohol
Belcher Pharmaceuticals, LLC
1 INDICATIONS AND USAGE Dehydrated alcohol injection is indicated to induce controlled cardiac septal infarction to improve exercise capacity in adult…
ELEVIDYS
DELANDISTROGENE MOXEPARVOVEC-ROKL
Sarepta Therapeutics, Inc.
1 INDICATIONS AND USAGE ELEVIDYS is indicated for the treatment of patients 4 years of age and older with Duchenne muscular dystrophy (DMD), who are a…
Ontak
denileukin diftitox
Eisai, Inc.
1 INDICATIONS AND USAGE LYMPHIR is indicated for the treatment of adult patients with relapsed or refractory Stage I-III cutaneous T-cell lymphoma (CT…
Lymphir
denileukin diftitox-cxdl
Citius Oncology, Inc.
1 INDICATIONS AND USAGE LYMPHIR is indicated for the treatment of adult patients with relapsed or refractory Stage I-III cutaneous T-cell lymphoma (CT…
XGEVA
denosumab
Amgen, Inc.
1 INDICATIONS AND USAGE Xgeva is a RANK ligand (RANKL) inhibitor indicated for: Prevention of skeletal-related events in patients with multiple myelom…
Desmopressin Acetate
DESMOPRESSIN ACETATE
Fresenius Kabi USA, LLC
1 INDICATIONS AND USAGE Desmopressin acetate injection is a vasopressin analog used for: Central Diabetes Insipidus - as antidiuretic replacement ther…
Austedo
deutetrabenazine
Teva Pharmaceuticals
1 INDICATIONS AND USAGE AUSTEDO XR ® and AUSTEDO ® are indicated in adults for the treatment of: chorea associated with Huntington’s disease [see Clin…
Dexamethasone
DEXAMETHASONE
Advanced Rx Pharmacy of Tennessee, LLC
INDICATIONS AND USAGE A l l ergic States: Control of severe or incapacitating a llergic conditions intractable to adequate trials of conventional trea…
Ozurdex
dexamethasone intravitreal implant
Allergan
1 INDICATIONS AND USAGE OZURDEX is a corticosteroid indicated for: The treatment of macular edema following branch retinal vein occlusion (BRVO) or ce…
Dexamethasone Sodium Phosphate
DEXAMETHASONE SODIUM PHOSPHATE
Hikma Pharmaceuticals USA Inc.
INDICATIONS A. Intravenous or intramuscular administration. When oral therapy is not feasible and the strength, dosage form, and route of administrati…
DEXMEDETOMIDINE HYDROCHLORIDE
DEXMEDETOMIDINE HYDROCHLORIDE
Sun Pharmaceuticals Industries, Inc.
1 INDICATIONS AND USAGE Dexmedetomidine hydrochloride is a alpha 2 -adrenergic receptor agonist indicated for: • Sedation of initially intubated and m…
Totect
dexrazoxane
Clinigen, Inc.
1 INDICATIONS AND USAGE Dexrazoxane for injection is indicated for reducing the incidence and severity of cardiomyopathy associated with doxorubicin a…
VALTOCO
diazepam (intranasal)
Neurelis Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE VALTOCO ® is indicated for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizu…
Vykat Xr
DIAZOXIDE CHOLINE
Soleno Therapeutics, Inc.
1 INDICATIONS AND USAGE VYKAT XR is indicated for the treatment of hyperphagia in adults and pediatric patients 4 years of age and older with Prader-W…
Durezol
difluprednate
Alcon Pharmaceuticals. Ltd.
1 INDICATIONS AND USAGE DUREZOL ® is a corticosteroid indicated for: • The treatment of inflammation and pain associated with ocular surgery. ( 1.1 ) …
Unituxin
dinutuximab
United Therapeutics Corporation
1 INDICATIONS AND USAGE Unituxin (dinutuximab) is indicated, in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleuki…
DIPHENOXYLATE HYDROCHLORIDE AND ATROPINE SULFATE
DIPHENOXYLATE HYDROCHLORIDE AND ATROPINE SULFATE
SpecGx LLC
DAWNZERA
DONIDALORSEN
Ionis Pharmaceuticals Inc.
1 INDICATIONS AND USAGE DAWNZERA™ is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 ye…