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.
Arikayce
liposomal amikacin
Insmed Incorporated
1 INDICATIONS AND USAGE LIMITED POPULATION: ARIKAYCE ® is indicated in adults, who have limited or no alternative treatment options, for the treatment…
AmBisome
Liposomal amphotericin B
Fujisawa USA, Inc.
INDICATIONS AND USAGE Am B isome is indicated for the following: • Empirical therapy for presumed fungal infection in febrile, neutropenic patients. •…
LISDEXAMFETAMINE DIMESYLATE
LISDEXAMFETAMINE DIMESYLATE
Camber Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE Lisdexamfetamine dimesylate capsules are indicated for the treatment of: Attention Deficit Hyperactivity Disorder (ADHD) in ad…
Breyanzi
lisocabtagene maraleucel
Juno Therapeutics, Inc.
1 INDICATIONS AND USAGE BREYANZI is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of: • adult patie…
Alomide Ophthalmic Solution
Lodoxamide tromethamine
Alcon Laboratories, Inc.
Treatment of ocular disorders referred to by the terms vernal keratoconjunctivitis, vernal conjunctivitis, vernal keratitis.…
Juxtapid
lomitapide
Chiesi Farmaceutici S.p.A.
1 INDICATIONS AND USAGE JUXTAPID is indicated as an adjunct to a low-fat diet and exercise and other low density lipoprotein cholesterol (LDL-C) thera…
Zokinvy
LONAFARNIB
Sentynl Therapeutics, Inc.
1 INDICATIONS AND USAGE ZOKINVY is indicated in patients 12 months of age and older with a body surface area (BSA) of 0.39 m 2 and above: To reduce th…
Skytrofa
lonapegsomatropin-tcgd
Ascendis Pharma Endocrinology Division A/S
1 INDICATIONS AND USAGE SKYTROFA (lonapegsomatropin-tcgd) is a human growth hormone indicated for the: Treatment of pediatric patients 1 year and olde…
Zynlonta
loncastuximab tesirine-lpyl
ADC Therapeutics SA
1 INDICATIONS AND USAGE ZYNLONTA is indicated for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more …
LORBRENA
Lorlatinib
Pfizer, Inc.
1 INDICATIONS AND USAGE LORBRENA ® is indicated for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors ar…
Lyfgenia
LOVOTIBEGLOGENE AUTOTEMCEL
Genetix Biotherapeutics Inc.
1 INDICATIONS AND USAGE LYFGENIA is indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-oc…
ORKAMBI
LUMACAFTOR AND IVACAFTOR
Vertex Pharmaceuticals Incorporated
1 INDICATIONS AND USAGE ORKAMBI is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 year and older who are homozygous for the F5…
Oxlumo
LUMASIRAN
Alnylam Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pedi…
Zepzelca
lurbinectedin
Jazz Pharmaceuticals Ireland Limited
1 INDICATIONS AND USAGE ZEPZELCA is an alkylating drug indicated: • in combination with atezolizumab or atezolizumab and hyaluronidase-tqjs, for the m…
Reblozyl
LUSPATERCEPT
Celgene Corporation
1 INDICATIONS AND USAGE REBLOZYL is an erythroid maturation agent indicated for the treatment of: • Anemia in adult patients with beta thalassemia who…
Lutathera
lutetium Lu 177 dotatate
Advanced Accelerator Applications
1 INDICATIONS AND USAGE LUTATHERA is indicated for the treatment of adult and pediatric patients 12 years and older with somatostatin receptor-positiv…
Macrelin
macimorelin acetate
Aeterna Zentaris GmbH
1 INDICATIONS AND USAGE MACRILEN is indicated for the diagnosis of adult growth hormone deficiency (AGHD). MACRILEN is a growth hormone (GH) secretago…
Opsumit
macitentan
Janssen
1 INDICATIONS AND USAGE OPSUMIT is an endothelin receptor antagonist (ERA) indicated for the treatment of pulmonary arterial hypertension (PAH, WHO Gr…
Opsynvi
MACITENTAN AND TADALAFIL
Actelion Pharmaceuticals US, Inc.
1 INDICATIONS AND USAGE OPSYNVI is a combination of macitentan, an endothelin receptor antagonist (ERA), and tadalafil, a phosphodiesterase 5 (PDE5) i…
Bronchitol
mannitol
Pharmaxis Europe Limited
1 INDICATIONS AND USAGE OSMITROL is indicated for: • The reduction of intracranial pressure and treatment of cerebral edema; • The reduction of elevat…
Livmarli
MARALIXIBAT CHLORIDE
Mirum Pharmaceuticals Inc.
1 INDICATIONS AND USAGE LIVMARLI is an ileal bile acid transporter (IBAT) inhibitor indicated for: • the treatment of cholestatic pruritus in patients…
Bupivilog Kit
MARCAINE, KENALOG, POVIDONE IODINE
Advanced Rx of Tennessee, LLC
1 INDICATIONS AND USAGE Bupivacaine Hydrochloride Injection is indicated in adults for the production of local or regional anesthesia or analgesia for…
Mlk Kit F2
MARCAINE, LIDOCAINE, KENALOG, POVIDONE IODINE
Asclemed USA, Inc.
1 INDICATIONS AND USAGE Bupivacaine Hydrochloride Injection is indicated in adults for the production of local or regional anesthesia or analgesia for…
Livtencity
maribavir
Takeda Pharmaceuticals U.S.A., Inc.
1 INDICATIONS AND USAGE LIVTENCITY ® is indicated for the treatment of adults and pediatric patients (12 years of age and older and weighing at least …
HYMPAVZI
MARSTACIMAB-HNCQ
Pfizer Laboratories Div Pfizer Inc
1 INDICATIONS AND USAGE HYMPAVZI is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric p…