FDA-Approved Treatments
Drugs with orphan designation or indications for rare diseases — 955 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.
Lantidra
donislecel-jujn
CellTrans Inc.
1 INDICATIONS AND USAGE LANTIDRA is an allogeneic pancreatic islet cellular therapy indicated for the treatment of adults with Type 1 diabetes who are…
Modeyso
dordaviprone
Chimerix, Inc.
1 INDICATIONS AND USAGE MODEYSO is indicated for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma har…
Pulmozyme
Dornase alfa
Genentech, Inc.
1 INDICATIONS AND USAGE PULMOZYME ® is indicated, in conjunction with standard therapies, for the management of pediatric and adult patients with cyst…
Kygevvi
doxecitine and doxribtimine
UCB, Inc.
1 INDICATIONS AND USAGE KYGEVVI is indicated for the treatment of thymidine kinase 2 deficiency (TK2d) in adults and pediatric patients with an age of…
Doxil
doxorubicin HCL liposome injection
Johnson & Johnson Pharmaceutical Research & Dev.
1 INDICATIONS AND USAGE Doxil liposomal infusion is an anthracycline topoisomerase inhibitor indicated for: • Ovarian cancer: After failure of platinu…
DOXYCYCLINE
DOXYCYCLINE
Strides Pharma Science Limited
INDICATIONS AND USAGE To reduce the development of drug-resistant bacteria and maintain effectiveness of doxycycline and other antibacterial drugs, do…
DRONABINOL
DRONABINOL
Ascend Laboratories, LLC
1 INDICATIONS AND USAGE DRONABINOL CAPSULES, USP are indicated in adults for the treatment of: anorexia associated with weight loss in patients with A…
NORTHERA
droxidopa
Lundbeck LLC
1 INDICATIONS AND USAGE NORTHERA is indicated for the treatment of orthostatic dizziness, lightheadedness, or the “feeling that you are about to black…
Dupixent
dupilumab
Regeneron Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE DUPIXENT is an interleukin-4 receptor alpha antagonist indicated: Atopic Dermatitis for the treatment of adult and pediatric p…
IMFINZI
durvalumab
AstraZeneca Pharmaceuticals LP
1 INDICATIONS AND USAGE IMFINZI is a programmed death-ligand 1 (PD-L1) blocking antibody indicated: • in combination with platinum-containing chemothe…
COPIKTRA
duvelisib
Secura Bio, Inc.
1 INDICATIONS AND USAGE COPIKTRA is indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or sm…
Kalbitor
ECALLANTIDE
Takeda Pharmaceuticals America, Inc.
1 INDICATIONS AND USAGE KALBITOR ® (ecallantide) is indicated for treatment of acute attacks of hereditary angioedema (HAE) in patients 12 years of ag…
SOLIRIS
ECULIZUMAB
Alexion Pharmaceuticals Inc.
1 INDICATIONS AND USAGE SOLIRIS is a complement inhibitor indicated for: the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to r…
EPYSQLI
ECULIZUMAB-AAGH
Teva Pharmaceuticals USA, Inc.
1 INDICATIONS AND USAGE EPYSQLI is a complement inhibitor indicated for: the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to r…
BKEMV
ECULIZUMAB-AEEB
Amgen Inc
1 INDICATIONS AND USAGE BKEMV is a complement inhibitor indicated for: The treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to red…
Radicava
edaravone
Tanabe Pharma America Inc.
1 INDICATIONS AND USAGE RADICAVA and RADICAVA ORS are indicated for the treatment of amyotrophic lateral sclerosis (ALS). RADICAVA and RADICAVA ORS ar…
Vyvgart
EFGARTIGIMOD ALFA
argenx US
1 INDICATIONS AND USAGE VYVGART is indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine rec…
Vyvgart Hytrulo
EFGARTIGIMOD ALFA AND HYALURONIDASE (HUMAN RECOMBINANT)
argenx US
1 INDICATIONS AND USAGE VYVGART HYTRULO is indicated for the treatment of adult patients with: generalized myasthenia gravis (gMG) who are anti-acetyl…
Iwilfin
eflornithine
USWM, LLC
1 INDICATIONS AND USAGE IWILFIN (eflornithine) is indicated to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma…
Kebilidi
eladocagene exuparvovec-tneq
PTC Therapeutics, Inc.
1 INDICATIONS AND USAGE KEBILIDI (eladocagene exuparvovec-tneq) is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatme…
Iqirvo
elafibranor
Ipsen Bioscience Inc.
1 INDICATIONS AND USAGE IQIRVO is indicated for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in …
Forzinity
elamipretide
Stealth Biotherapeutics, Inc.
1 INDICATIONS AND USAGE FORZINITY is indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg.…
Revcovi
elapegademase-lvlr
Chiesi USA Inc.
1 INDICATIONS AND USAGE REVCOVI is indicated for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and ad…
Trikafta
elexacaftor/tezacaftor/ivacaftor
Vertex Pharmaceuticals
1 INDICATIONS AND USAGE TRIKAFTA is indicated for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one F508d…