FDA-Approved Treatments
Drugs with orphan designation or indications for rare diseases — 968 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.
Amatine
Midodrine HCl
Takeda Development Center Americas, Inc.
Treatment of symptomatic orthostatic hypotension.…
Korlym
mifepristone
Corcept Therapeutics, Inc.
1 INDICATIONS AND USAGE KORLYM (mifepristone) is a cortisol receptor blocker indicated to control hyperglycemia secondary to hypercortisolism in adult…
Galafold
MIGALASTAT HYDROCHLORIDE
Amicus Therapeutics US, LLC
1 INDICATIONS AND USAGE GALAFOLD is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase al…
OPFOLDA
MIGLUSTAT
AMICUS THERAPEUTICS US, LLC
1 INDICATIONS AND USAGE OPFOLDA is indicated, in combination with Pombiliti, for the treatment of adult patients with late-onset Pompe disease (lysoso…
IMPAVIDO
miltefosine
Knight Therapeutics (USA)
1 INDICATIONS AND USAGE IMPAVIDO (miltefosine) capsules are indicated in adults and adolescents ≥12 years of age weighing ≥ 30 kg for the treatment of…
KYNAMRO
mipomersen
Kastle Therapeutics, LLC
Adjunct to lipid-lowering medications and diet to reduce low density lipoprotein-cholesterol (LDL-C), apolipoprotein B (apo B), total cholesterol (TC)…
Gomekli
mirdametinib
SpringWorks Therapeutics (EMD Serono / Merck KGaA)
1 INDICATIONS AND USAGE GOMEKLI is indicated for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 …
Elahere
mirvetuximab soravtansine-gynx
ImmunoGen, Inc.
1 INDICATIONS AND USAGE ELAHERE ® is indicated for the treatment of adult patients with folate receptor-alpha (FRα) positive, platinum-resistant epith…
AQVESME
MITAPIVAT
Agios Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE AQVESME is indicated for the treatment of anemia in adults with alpha- or beta-thalassemia. AQVESME is a pyruvate kinase activ…
Jelmyto
mitomycin
UroGen Pharma, Ltd.
1 INDICATIONS AND USAGE JELMYTO ® is indicated for the treatment of adult patients with low-grade Upper Tract Urothelial Cancer (LG-UTUC). JELMYTO is …
Mitosol
mitomycin-C
Glaukos Corporation
1 INDICATIONS AND USAGE Mitosol ® is an antimetabolite indicated for use as an adjunct to ab externo glaucoma surgery. Mitosol ® is an antimetabolite …
Novantrone
Mitoxantrone
Serono, Inc.
INDICATIONS AND USAGE Mitoxantrone injection, USP (concentrate) is indicated for reducing neurologic disability and/or the frequency of clinical relap…
Exkivity
mobocertinib
Takeda Development Center Americas, Inc.
Treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 2…
Provigil
Modafinil
Cephalon, Inc.
1 INDICATIONS AND USAGE PROVIGIL is indicated to improve wakefulness in adult patients with excessive sleepiness associated with narcolepsy, obstructi…
POTELIGEO
mogamulizumab
Kyowa Kirin Pharmaceutical Development Inc.
1 INDICATIONS AND USAGE POTELIGEO is indicated for the treatment of adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndro…
Ojjaara
MOMELOTINIB
GlaxoSmithKline LLC
1 INDICATIONS AND USAGE OJJAARA is indicated for the treatment of intermediate or high-risk myelofibrosis (MF), including primary MF or secondary MF […
Infumorph
Morphine sulfate concentrate (preservative free)
Elkins-Sinn, Inc.
1 INDICATIONS AND USAGE INFUMORPH is for use in continuous microinfusion devices and indicated only for intrathecal or epidural infusion in the manage…
Lunsumio
mosunetuzumab-axgb
Genentech, Inc.
1 INDICATIONS AND USAGE LUNSUMIO is a bispecific CD20-directed CD3 T-cell engager indicated for the treatment of adult patients with relapsed or refra…
Aphexda
motixafortide
Ayrmid Pharma Ltd.
1 INDICATIONS AND USAGE APHEXDA is indicated in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for c…
LUMOXITI
moxetumomab pasudotox-tdfk
AstraZeneca Pharmaceuticals LP
LUMOXITI is indicated for the treatment of adult patients with relapsed or refractory hairy cell leukemia (HCL) who received at least two prior system…
M.V.I.-12
multi-vitamin infusion without vitamin K
Mayne Pharma (USA) Inc.
Prevention of vitamin deficiency and thrombormbolic complications in people receiving home parenteral nutrition who also receive warfarin-type anticoa…
MYCOPHENOLATE MOFETIL
MYCOPHENOLATE MOFETIL
Aurobindo Pharma Limited
1 INDICATIONS AND USAGE Mycophenolate mofetil (MMF) is indicated for the prophylaxis of organ rejection, in adult and pediatric recipients 3 months of…
Synarel Nasal Solution
Nafarelin acetate
Syntex (USA), Inc.
INDICATIONS AND USAGE FOR CENTRAL PRECOCIOUS PUBERTY (For Endometriosis, See Reverse Side ) SYNAREL is indicated for treatment of central precocious p…
Yartemlea
narsoplimab-wuug
Omeros Corporation
1 INDICATIONS AND USAGE YARTEMLEA is indicated for the treatment of adult and pediatric patients 2 years of age and older with hematopoietic stem cell…
Yuviwel
navepegritide
Ascendis Pharma Growth Disorders A/S
to increase linear growth in pediatric patients 2 years of age and older with achondroplasia with open epiphyses…
DANYELZA
naxitamab-gqgk
Y-mAbs Therapeutics, Inc.
in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of pediatric patients 1 year of age and older and adu…