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.
ADAKVEO
CRIZANLIZUMAB
Novartis Pharmaceuticals Corporation
1 INDICATIONS AND USAGE ADAKVEO ® is indicated to reduce the frequency of vaso-occlusive crises (VOCs) in adults and pediatric patients aged 16 years …
Xalkori
crizotinib
Pfizer, Inc.
1 INDICATIONS AND USAGE XALKORI is a kinase inhibitor indicated for the treatment of • adult patients with metastatic non-small cell lung cancer (NSCL…
Gastrocrom
Cromolyn sodium
Fisons Corporation
INDICATIONS AND USAGE GASTROCROM is indicated in the management of patients with mastocytosis. Use of this product has been associated with improvemen…
Anavip
crotalidae immune F(ab')2 (equine)
Rare Disease Therapeutics, Inc.
Management of adult and pediatric patients with North American rattlesnake envenomation ||| For the management of adult and pediatric patients with No…
Piasky
CROVALIMAB
Genentech, Inc.
1 INDICATIONS AND USAGE PIASKY is indicated for the treatment of adult and pediatric patients 13 years and older with paroxysmal nocturnal hemoglobinu…
Verkazia
cyclosporine
Harrow Eye, LLC
1 INDICATIONS AND USAGE Verkazia ophthalmic emulsion is indicated for the treatment of vernal keratoconjunctivitis (VKC) in children and adults. Verka…
Cystagon
Cysteamine
Mylan Laboratories, Inc.
INDICATIONS AND USAGE CYSTAGON ® is indicated for the management of nephropathic cystinosis in children and adults.…
PROCYSBI
cysteamine enteric coated
Horizon Therapeutics USA, Inc.
1 INDICATIONS AND USAGE PROCYSBI is indicated for the treatment of nephropathic cystinosis in adults and pediatric patients 1 year of age and older. P…
Cystaran
Cysteamine hydrochloride
Leadiant Biosciences, Inc.
1 INDICATIONS AND USAGE CYSTARAN ® is a cystine-depleting agent indicated for the treatment of corneal cystine crystal accumulation in patients with c…
DepoCyt
Cytarabine liposomal
Pacira Pharmaceuticals, Inc.
Intrathecal treatment of lymphomatous meningitis.…
VYXEOS
Cytarabine:daunorubicin liposome injection; daunorubicin and cytarabine (liposome); daunorubicin and cytarabine liposome for injection
Celator Pharmaceuticals, Inc. (a Jazz Pharmaceuticals Company)
treatment of newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in adults and pediatr…
CytoGam
Cytomegalovirus immune globulin (human)
CSL Behring LLC
#1) Attenuation of primary CMV disease associated with kidney transplant recipients who are seronegative for CMV and who receive a kidney from a CMV s…
TAFINLAR
dabrafenib
Novartis Pharmaceuticals Corp.
1 INDICATIONS AND USAGE TAFINLAR is a kinase inhibitor indicated as a single agent for the treatment of patients with unresectable or metastatic melan…
Tafinlar Capsules and Mekinist Tablets
dabrafenib and trametinib
Novartis Pharmaceuticals Corporation
MEKINIST is indicated, in combination with dabrafenib, for the treatment of patients with locally advanced or metastatic anaplastic thyroid cancer (AT…
Zenapax
Daclizumab
Hoffmann-La Roche, Inc.
Prophylaxis of acute organ rejection in patients receiving renal transplants, to be used as a part of an immunosuppressive regimen that includes cyclo…
VIZIMPRO
dacomitinib
Pfizer, Inc.
1 INDICATIONS AND USAGE VIZIMPRO is indicated for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) with epiderm…
Ampyra
dalfampridine
Acorda Therapeutics
1 INDICATIONS AND USAGE AMPYRA is indicated as a treatment to improve walking in adult patients with multiple sclerosis (MS). This was demonstrated by…
Danazol
DANAZOL
Chartwell RX, LLC.
INDICATIONS AND USAGE Endometriosis. Danazol capsules are indicated for the treatment of endometriosis amenable to hormonal management. Hereditary Ang…
Voydeya
DANICOPAN
Alexion Pharmaceuticals Inc.
1 INDICATIONS AND USAGE VOYDEYA is indicated as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adul…
RYANODEX
dantrolene sodium suspension for injection
Eagle Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE RYANODEX ® is indicated for the: Treatment of malignant hyperthermia in conjunction with appropriate supportive measures [see …
DAPAGLIFLOZIN AND METFORMIN HYDROCHLORIDE
DAPAGLIFLOZIN AND METFORMIN HYDROCHLORIDE
Lupin Pharmaceuticals, Inc.
Darzalex
daratumumab
Janssen Biotech, Inc.
1 INDICATIONS AND USAGE DARZALEX FASPRO is a combination of daratumumab, a CD38-directed cytolytic antibody, and hyaluronidase, an endoglycosidase, in…
Darzalex Faspro
DARATUMUMAB AND HYALURONIDASE-FIHJ (HUMAN RECOMBINANT)
Janssen Biotech, Inc.
1 INDICATIONS AND USAGE DARZALEX FASPRO is a combination of daratumumab, a CD38-directed cytolytic antibody, and hyaluronidase, an endoglycosidase, in…
Phyrago
dasatinib
Handa Therapeutics, LLC
1. INDICATIONS AND USAGE PHYRAGO is indicated for the treatment of adult patients with newly diagnosed Philadelphia chromosome-positive (Ph+) chronic …
DaunoXome
Daunorubicin citrate liposome injection
NeXstar Pharmaceuticals, Inc.
First line cytotoxic therapy for advanced, HIV related Kaposi's sarcoma.…
Daxxify
daxibotulinumtoxinA-lanm
Revance Therapeutics, Inc.
1 INDICATIONS AND USAGE DAXXIFY is an acetylcholine release inhibitor and neuromuscular-blocking agent indicated for: Temporary improvement in the app…
Decitabine
DECITABINE
BluePoint Laboratories
1 INDICATIONS AND USAGE Decitabine for Injection is indicated for treatment of adult patients with myelodysplastic syndromes (MDS) including previousl…
Deferasirox
DEFERASIROX
MSN LABORATORIES PRIVATE LIMITED
1 INDICATIONS AND USAGE Deferasirox tablets are an iron chelator indicated for the treatment of chronic iron overload due to blood transfusions in pat…