FDA-Approved Treatments

Drugs with orphan designation or indications for rare diseases — 970 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.

RINGERS

SODIUM CHLORIDE, POTASSIUM CHLORIDE, AND CALCIUM CHLORIDE

ICU Medical, Inc.

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RINGERS

SODIUM CHLORIDE, POTASSIUM CHLORIDE, AND CALCIUM CHLORIDE

ICU Medical, Inc.

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Orphan⚠️ Black Box

Nithiodote

sodium nitrite and sodium thiosulfate

Hope Pharmaceuticals

1 INDICATIONS AND USAGE NITHIODOTE is indicated for the treatment of acute cyanide poisoning that is judged to be serious or life-threatening. When th

Cyanide poisoning
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Orphan⚠️ Black Box

Lumryz

sodium oxybate

Flamel Ireland Limited dba Avadel Ireland

1 INDICATIONS AND USAGE LUMRYZ is indicated for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older

NarcolepsyNarcolepsy type 2Idiopathic hypersomnia
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Orphan

Sodium Phenylacetate And Sodium Benzoate

SODIUM PHENYLACETATE AND SODIUM BENZOATE

Zydus Pharmaceuticals USA Inc.

1 INDICATIONS AND USAGE Sodium Phenylacetate and Sodium Benzoate Injection is a nitrogen binding agent indicated as adjunctive therapy for the treatme

Hypermethioninemia encephalopathy due to adenosine kinase deficiencyView details →
Orphan

Sodium Phenylbutyrate

SODIUM PHENYLBUTYRATE

GLENMARK PHARMACEUTICALS INC., USA

INDICATIONS AND USAGE Sodium phenylbutyrate tablets are indicated as adjunctive therapy in the chronic management of patients with urea cycle disorder

Ornithine transcarbamylase deficiencyCitrullinemia type ICarbamoyl-phosphate synthetase 1 deficiency
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Orphan

Relyvrio

sodium phenylbutyrate and taurursodiol

Amylyx Pharmaceuticals, Inc.

Treatment of amyotrophic lateral sclerosis (ALS) in adults

Amyotrophic Lateral Sclerosis (ALS)View details →
Orphan

Sodium Phenylbutyrate

SODIUM PHENYLBUTYRATE TABLETS, 500 MG

ENDO USA, Inc.

INDICATIONS AND USAGE Sodium Phenylbutyrate Tablets is indicated as adjunctive therapy in the chronic management of patients with urea cycle disorders

Primary immunodeficiency with natural-killer cell deficiency and adrenal insufficiencyUrea Cycle DisorderOrnithine transcarbamylase deficiencyCitrullinemia type IRare familial disorder with hypertrophic cardiomyopathyCarbamoyl-phosphate synthetase 1 deficiency
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Orphan

SUPREP Bowel Prep Kit

sodium sulfate, potassium sulfate, and magnesium sulfate

Braintree Laboratories, Inc.

1 INDICATIONS & USAGE Sodium sulfate, potassium sulfate and magnesium sulfate oral solution is indicated for cleansing of the colon as a preparation f

Secondary interstitial lung disease in childhood and adulthoodView details →
Orphan

Pedmark

sodium thiosulfate

Fennec Pharmaceuticals, Inc.

1 INDICATIONS AND USAGE PEDMARK is indicated to reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1 month of age and olde

Recurrent hepatitis C virus induced liver disease in liver transplant recipientsView details →
Orphan⚠️ Black Box

Sovaldi

sofosbuvir

Gilead Sciences, Inc.

1 INDICATIONS AND USAGE SOVALDI is a hepatitis C virus (HCV) nucleotide analog NS5B polymerase inhibitor indicated for the treatment of: Adult patient

Congenital varicella syndromeChronic Epstein-Barr virus infection syndrome
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Orphan⚠️ Black Box

Epclusa

sofosbuvir and velpatasvir

Gilead Sciences, Inc.

1 INDICATIONS AND USAGE EPCLUSA is indicated for the treatment of adults and pediatric patients 3 years of age and older with chronic hepatitis C viru

Congenital varicella syndromeBartonella bacilliformis infectionChronic Epstein-Barr virus infection syndrome
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Orphan

Sunosi

Solriamfetol

Axsome Malta, Ltd.

1 INDICATIONS AND USAGE SUNOSI is indicated to improve wakefulness in adult patients with excessive daytime sleepiness associated with narcolepsy or o

NarcolepsyOBSOLETE: Primary pigmented nodular adrenocortical disease
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Orphan

Sogroya

somapacitan-beco

Novo Nordisk Pharmaceuticals

1 INDICATIONS AND USAGE SOGROYA is indicated for the treatment of pediatric patients aged 2.5 years and older with: • Growth failure due to inadequate

Growth and developmental delay-hypotonia-vision impairment-lactic acidosis syndromeRare disorder due to inadequate sharing of the placentaShort stature-intellectual disability-eye anomalies-cleft lip/palate syndromeOtomandibular dysplasia associated with monogenic syndromesIdiopathic steroid-resistant nephrotic syndromeLaron syndrome
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Orphan

Protropin

Somatrem for injection

Genentech, Inc.

Growth hormone insensitivity syndrome
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Orphan

Ngenla

somatrogon-ghla

Pfizer Ireland Pharmaceuticals

1 INDICATIONS AND USAGE NGENLA is indicated for the treatment of pediatric patients aged 3 years and older who have growth failure due to an inadequat

Non-acquired isolated growth hormone deficiencyRare disorder due to inadequate sharing of the placenta
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Orphan

Norditropin

SOMATROPIN

Novo Nordisk

1 INDICATIONS AND USAGE NORDITROPIN is a recombinant human growth hormone indicated for: • Pediatric : Treatment of pediatric patients with growth fai

Prader-Willi syndrome
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Orphan

Zorbtive

Somatropin (r-DNA)

EMD Serono, Inc.

Treatment of short bowel syndrome in patients receiving specialized nutritional support

CADINS diseaseView details →
Orphan

Nutropin Depot

Somatropin (rDNA origin)

Genentech, Inc.

Long-term treatment of growth failure due to a lack of adequate endogenous growth hormone secetion for once- or twice-a-month administration.

Acute infantile liver failure due to synthesis defect of mtDNA-encoded proteinsLaron syndrome
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Orphan

Serostim

Somatropin for injection

EMD Serono, Inc.

1 INDICATIONS AND USAGE SEROSTIM (somatropin) is indicated for the treatment of HIV patients with wasting or cachexia to increase lean body mass and b

AIDS wasting syndrome
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Orphan

Nexavar

sorafenib

Bayer HealthCare Pharmaceuticals, Inc.

1 INDICATIONS AND USAGE NEXAVAR is a kinase inhibitor indicated for the treatment of • Unresectable hepatocellular carcinoma ( 1.1 ) • Advanced renal

Pediatric hepatocellular carcinomaAdult hepatocellular carcinomaRenal cell carcinoma
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Orphan

So-Aqueous

sotalol (IV)

AltaThera Pharmaceuticals LLC

For use as a substitute for oral sotalol in patients who are unable to take sotalol orally [oral sotalol is indicated for maintenance of normal sinus

Catecholaminergic polymorphic ventricular tachycardiaHereditary atrial fibrillation
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Orphan⚠️ Black Box

Betapace

Sotalol HCl

Berlex Laboratories, Inc.

1 INDICATIONS AND USAGE Betapace/Betapace AF is an antiarrhythmic indicated for: the treatment of life-threatening ventricular arrhythmias ( 1.1 ) the

Double outlet right ventricle with subpulmonary ventricular septal defectView details →

WINREVAIR

SOTATERCEPT-CSRK

Merck Sharp & Dohme LLC

1 INDICATIONS AND USAGE WINREVAIR™ is indicated for the treatment of adults with pulmonary arterial hypertension (PAH, Group 1 pulmonary hypertension)

Pulmonary arterial hypertension
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Orphan

Lumakras

sotorasib

Amgen Inc.

1 INDICATIONS AND USAGE LUMAKRAS is an inhibitor of the RAS GTPase family indicated for: KRAS G12C-mutated Locally Advanced or Metastatic Non-Small Ce

Hereditary nonpolyposis colon cancerNON RARE IN EUROPE: Non-small cell lung cancer
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Orphan⚠️ Black Box

Filspari

sparsentan

Travere Therapeutics

1 INDICATIONS AND USAGE FILSPARI is indicated to slow kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) who are at ri

Immunoglobulin A nephropathyIgA Nephropathy
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Orphan

Spevigo

spesolimab-sbzo

Boehringer Ingelheim Pharmaceuticals, Inc. (BI)

1 INDICATIONS AND USAGE SPEVIGO is indicated for the treatment of generalized pustular psoriasis (GPP) in adults and pediatric patients 12 years of ag

Generalized pustular psoriasis
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Orphan

Steritalc

Sterile talc

Novatech SA

1.INDICATIONS AND USAGE 1.INDICATIONS AND USAGE 1.1 Malignant Pleural Effusion STERITALC is indicated to decrease the recurrence of malignant pleural

Familial spontaneous pneumothoraxView details →
Orphan

Sclerosol Intrapleural Aerosol

Sterile talc powder

Sciarra Laboratories, Inc.

Prevention of recurrence of malignant pleural effusions in symptomatic patients.

Malignant peritoneal mesothelioma
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Orphan

Diacomit

stiripentol

Biocodex

Treatment of seizures associated with Dravet syndrome (DS) in patients taking clobazam who are 6 months of age and older and weighing 7 kg or more |||

Dravet syndrome
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