FDA-Approved Treatments

Drugs with orphan designation or indications for rare diseases — 955 total

Treatment data is sourced from FDA drug labels, DailyMed, and openFDA.
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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.

Orphan⚠️ Black Box

Zevalin

ibritumomab tiuxetan

Acrotech Biopharma LLC

1 INDICATIONS AND USAGE Zevalin is a CD20-directed radiotherapeutic antibody administered as part of the Zevalin therapeutic regimen indicated for the

Indolent B-cell non-Hodgkin lymphomaOBSOLETE: Primary intraocular lymphomaAggressive B-cell non-Hodgkin lymphoma
View details →
Orphan

IMBRUVICA

ibrutinib

Pharmacyclics, LLC

1 INDICATIONS AND USAGE IMBRUVICA is a kinase inhibitor indicated for the treatment of: Adult patients with chronic lymphocytic leukemia (CLL)/Small l

Splenic marginal zone lymphomaWaldenström MacroglobulinemiaChronic graft versus host diseaseB-lymphoblastic leukemia/lymphoma with t(17;19)B-cell chronic lymphocytic leukemia
View details →
Orphan

NeoProfen

Ibuprofen lysine

Lundbeck, Inc.

1 INDICATIONS AND USAGE NeoProfen is indicated to close a clinically significant patent ductus arteriosus (PDA) in premature infants weighing between

Arterial duct anomaly
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Orphan

SAJAZIR

ICATIBANT

Cycle Pharmaceuticals Ltd-Uk

1 INDICATIONS AND USAGE SAJAZIR (icatibant) injection is indicated for the treatment of acute attacks of hereditary angioedema (HAE) in adults 18 year

Hereditary angioedema
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Orphan

ICATIBANT

ICATIBANT ACETATE

Bryant Ranch Prepack

1 INDICATIONS AND USAGE Icatibant injection is indicated for the treatment of acute attacks of hereditary angioedema (HAE) in adults 18 years of age a

Hereditary angioedema
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Orphan

Extraneal (with 7.5% Icodextrin) Peritoneal Dialysis Solutio

Icodextrin 7.5% with Electrolytes Peritoneal Dialysis Solution

Baxter Healthcare Corporation

For use as a single daily exchange for the long (8-16 hour) dwell during continuous ambulatory peritoneal dialysis (CAPD) or automated peritoneal dial

Off-periods in Parkinson disease not responding to oral treatmentChronic intestinal failure
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Orphan⚠️ Black Box

Idamycin

Idarubicin HCl for injection

Adria Laboratories, Inc.

1 INDICATIONS AND USAGE IDAMYCIN PFS is indicated for the treatment of adult patients with acute myeloid leukemia (AML) as a component of a combinatio

Therapy related acute myeloid leukemia and myelodysplastic syndromeAcute megakaryoblastic leukemia
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Orphan⚠️ Black Box

Abecma

idecabtagene vicleucel

Celgene Corporation, a Bristol-Myers Squibb Company

1 INDICATIONS AND USAGE ABECMA is indicated for the treatment of adult patients with relapsed or refractory multiple myeloma after two or more prior l

Multiple myeloma
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Orphan⚠️ Black Box

Zydelig

idelalisib

Gilead Sciences, Inc.

1 INDICATIONS AND USAGE Zydelig is indicated, in combination with rituximab, for the treatment of patients with relapsed chronic lymphocytic leukemia

Follicular lymphomaT-cell prolymphocytic leukemia
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Orphan⚠️ Black Box

ELAPRASE

IDURSULFASE

Takeda Pharmaceuticals America, Inc.

1 INDICATIONS AND USAGE ELAPRASE is indicated for patients with Hunter syndrome (Mucopolysaccharidosis II, MPS II). ELAPRASE has been shown to improve

Mucopolysaccharidosis type 2
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Orphan⚠️ Black Box

Ifex

Ifosfamide

Bristol-Myers Squibb Pharmaceutical Research Institute

1 INDICATIONS AND USAGE IFEX is indicated for use in adults in combination with certain other approved antineoplastic agents for third-line chemothera

Rare tumor of intestineView details →
Orphan

Aurlumyn

iloprost

BTG International Inc.

1 INDICATIONS AND USAGE AURLUMYN is a prostacyclin mimetic indicated for the treatment of severe frostbite in adults to reduce the risk of digit amput

Testicular teratomaSevere phosphoribosylpyrophosphate synthetase superactivity
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Orphan

Ventavis

Iloprost inhalation solution

CoTherix, Inc.

Treatment of pulmonary arterial hypertension (WHO Group I) in patients with NYHA Class III or IV symptoms

Pulmonary arterial hypertension
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Orphan

Imatinib Mesylate

IMATINIB

Mylan Institutional Inc

1 INDICATIONS AND USAGE Imatinib mesylate tablets are a kinase inhibitor indicated for the treatment of: Newly diagnosed adult and pediatric patients

Gastrointestinal stromal tumorB-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2)Precursor T-cell acute lymphoblastic leukemiaDermatofibrosarcoma protuberans
View details →
Orphan

Imatinib Mesylate

IMATINIB MESYLATE

Apotex Corp

1 INDICATIONS AND USAGE Imatinib mesylate is a kinase inhibitor indicated for the treatment of: • Newly diagnosed adult and pediatric patients with Ph

Acute myeloid leukemia and myelodysplastic syndromes related to topoisomerase type 2 inhibitorIdiopathic chronic eosinophilic pneumoniaDermatofibrosarcoma protuberansMyelodysplastic/myeloproliferative diseasePrecursor T-cell acute lymphoblastic leukemiaGastrointestinal stromal tumorB-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2)Aggressive systemic mastocytosis
View details →
Orphan

Imkeldi

IMATINIB ORAL

Shorla Oncology Inc.,

1 INDICATIONS AND USAGE Imkeldi is a kinase inhibitor indicated for the treatment of: Newly diagnosed adult and pediatric patients with Philadelphia c

Dermatofibrosarcoma protuberansB-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2)Precursor T-cell acute lymphoblastic leukemiaMyelodysplastic/myeloproliferative disease
View details →
Orphan

Rytelo

IMETELSTAT SODIUM

Geron Corporation

1. INDICATIONS AND USAGE RYTELO is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with

Myelodysplastic syndromeMyelodysplastic syndrome associated with isolated del(5q) chromosome abnormality
View details →
Orphan⚠️ Black Box

Cerezyme

IMIGLUCERASE

Genzyme Corporation

1 INDICATIONS AND USAGE CEREZYME is indicated for the treatment of non-central nervous system (CNS) manifestations of Type 1 or Type 3 Gaucher disease

BENTA diseaseGaucher disease
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Orphan

Gamunex-C

Immune Globulin (Human)

Grifols Therapeutics, Inc.

INDICATIONS AND USAGE Nabi-HB, Hepatitis B Immune Globulin (Human), is indicated for treatment of acute exposure to blood containing HBsAg, perinatal

Chronic inflammatory demyelinating polyneuropathyView details →
Orphan

Gammagard Liquid

immune globulin infusion (human)

Takeda Pharmaceuticals U.S.A., Inc.

Maintenance therapy to improve muscle strength and disability in adult patients with Multifocal Motor Neuropathy (MMN).

Multifocal motor neuropathy
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Orphan

HyQvia

Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase

Takeda Development Center Americas, Inc.

treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) as maintenance therapy to prevent relapse of neuromuscular disability and impair

Chronic inflammatory demyelinating polyneuropathy
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Orphan⚠️ Black Box

Gamimune N

Immune globulin intravenous, human

Bayer Corporation

1 INDICATIONS AND USAGE BIVIGAM is an Immune Globulin Intravenous (Human), 10% Liquid, indicated for the treatment of adults and pediatric patients 2

Infective dermatitis associated with HTLV-1
View details →
Orphan

Hizentra

Immune Globulin Subcutaneous (Human), 20% Liquid

CSL Behring

Indicated for the treatment of adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP) as maintenance therapy to prevent relapse

Chronic inflammatory demyelinating polyneuropathy
View details →

ITOVEBI

INAVOLISIB

Genentech, Inc.

View details →
Orphan

Leqvio

inclisiran

Novartis Pharmaceuticals Corporation

1 INDICATIONS AND USAGE LEQVIO ® is indicated as an adjunct to diet and exercise to reduce low-density lipoprotein cholesterol (LDL-C) in: adults with

Homozygous familial hypercholesterolemia
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⚠️ Black Box

XEOMIN

INCOBOTULINUMTOXINA

Merz Pharmaceuticals, LLC

1 INDICATIONS AND USAGE XEOMIN is an acetylcholine release inhibitor and neuromuscular blocking agent indicated for the treatment or improvement of: C

X-linked Ehlers-Danlos syndromeView details →
Orphan

Uplizna

INEBILIZUMAB

Horizon Therapeutics USA, Inc.

1 INDICATIONS AND USAGE UPLIZNA is a CD19-directed cytolytic antibody indicated for the treatment of: Neuromyelitis optica spectrum disorder (NMOSD) i

Neuromyelitis optica spectrum disorderIgG4-related diseaseTransient neonatal myasthenia gravis
View details →
Orphan

Truseltiq

infigratinib

QED Therapeutics, Inc.

Treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2

Combined hepatocellular carcinoma and cholangiocarcinoma
View details →
Orphan⚠️ Black Box

Remicade

infliximab

Janssen Biotech, Inc.

1 INDICATIONS AND USAGE REMICADE is a tumor necrosis factor (TNF) blocker indicated for: Crohn's Disease : reducing signs and symptoms and inducing an

NON RARE IN EUROPE: Ulcerative colitisBartonella bacilliformis infectionPediatric multiple sclerosisPsoriasis-related juvenile idiopathic arthritisFilariasisPediatric-onset Graves diseaseNON RARE IN EUROPE: Ankylosing spondylitisPeroxisomal disease with epilepsyJuvenile idiopathic arthritis
View details →
Orphan

Tegsedi

inotersen

Ionis/AstraZeneca

Indicated for polyneuropathy of hATTR amyloidosis in adults.

Hereditary ATTR amyloidosis
View details →
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