FDA-Approved Treatments
Drugs with orphan designation or indications for rare diseases — 970 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.
KANUMA
SEBELIPASE ALFA
Alexion Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE KANUMA ® is indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency. KANUMA ® is a …
EKTERLY
SEBETRALSTAT
KalVista Pharmaceuticals Ltd
1 INDICATIONS AND USAGE EKTERLY® is indicated for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 1…
Livdelzi
seladelpar
Gilead Sciences, Inc.
1 INDICATIONS AND USAGE LIVDELZI is indicated for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) i…
Uptravi
selexipag
Janssen
1 INDICATIONS AND USAGE UPTRAVI is a prostacyclin receptor agonist indicated for the treatment of pulmonary arterial hypertension (PAH, WHO Group I) t…
XPOVIO
selinexor
Karyopharm Therapeutics, Inc.
1 INDICATIONS AND USAGE XPOVIO is a nuclear export inhibitor indicated: In combination with bortezomib and dexamethasone for the treatment of adult pa…
RETEVMO
selpercatinib
Loxo Oncology Inc., a wholly owned subsidiary of Eli Lilly and Company
1 INDICATIONS AND USAGE RETEVMO ® is a kinase inhibitor indicated for the treatment of: Adult patients with locally advanced or metastatic non-small c…
Koselugo
selumetinib
AstraZeneca / Alexion
1 INDICATIONS AND USAGE KOSELUGO is indicated for the treatment of adult and pediatric patients 1 year of age and older with neurofibromatosis type 1 …
WEGOVY
SEMAGLUTIDE
Novo Nordisk
1 INDICATIONS AND USAGE WEGOVY injection is indicated in combination with a reduced calorie diet and increased physical activity: • To reduce the risk…
Sephience
SEPIAPTERIN
PTC Therapeutics Inc.
1 INDICATIONS AND USAGE SEPHIENCE is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and old…
IMCIVREE
SETMELANOTIDE
Rhythm Pharmaceuticals, Inc
1 INDICATIONS AND USAGE IMCIVREE is indicated to reduce excess body weight and maintain weight reduction long term in adults and pediatric patients ag…
Hyrnuo
sevabertinib
Bayer HealthCare Pharmaceuticals Inc.
1 INDICATIONS AND USAGE HYRNUO is indicated for the treatment of adult patients with locally advanced or metastatic non-squamous non-small cell lung c…
Voyxact
sibeprenlimab
Otsuka
1 INDICATIONS AND USAGE VOYXACT is indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease prog…
Sildenafil
SILDENAFIL
Asclemed USA, Inc.
1 INDICATIONS & USAGE Sildenafil tablets are indicated for the treatment of pulmonary arterial hypertension (WHO Group I) in adults to improve exercis…
Sildenafil
SILDENAFIL CITRATE
ANI Pharmaceuticals, Inc.
1 INDICATIONS & USAGE Adults Sildenafil for oral suspension is indicated for the treatment of pulmonary arterial hypertension (PAH) (World Health Orga…
Sildenafil
SILDENAFIL POWDER,
Camber Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE Adults Sildenafilfororal suspension is indicated for the treatment of pulmonary arterial hypertension (PAH) (World Health Orga…
SYLVANT
siltuximab
Recordati Rare Diseases Inc.
1 INDICATIONS AND USAGE SYLVANT is indicated for the treatment of patients with multicentric Castleman's disease (MCD) who are human immunodeficiency …
Hyftor
SIROLIMUS
Nobelpharma America, LLC
1 INDICATIONS AND USAGE HYFTOR is indicated for the treatment of facial angiofibroma associated with tuberous sclerosis in adults and pediatric patien…
Fyarro
sirolimus protein-bound particles for injectable suspension (albumin-bound)
Aadi Bioscience, Inc.
1 INDICATIONS AND USAGE FYARRO ™ is indicated for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascu…
RINGERS
SODIUM CHLORIDE, POTASSIUM CHLORIDE, AND CALCIUM CHLORIDE
ICU Medical, Inc.
1 INDICATIONS AND USAGE Ringer’s Injection is indicated for use as a source of water and electrolytes in adults and pediatric patients. Ringer’s Injec…
RINGERS
SODIUM CHLORIDE, POTASSIUM CHLORIDE, AND CALCIUM CHLORIDE
ICU Medical, Inc.
1 INDICATIONS AND USAGE Ringer’s Injection is indicated for use as a source of water and electrolytes in adults and pediatric patients. Ringer’s Injec…
RINGERS
SODIUM CHLORIDE, POTASSIUM CHLORIDE, AND CALCIUM CHLORIDE
ICU Medical, Inc.
1 INDICATIONS AND USAGE Ringer’s Injection is indicated for use as a source of water and electrolytes in adults and pediatric patients. Ringer’s Injec…