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.
Rifater
Rifampin, isoniazid, pyrazinamide
Hoechst Marion Roussel
Short-course treatment of tuberculosis.…
Priftin
Rifapentine
Hoechst Marion Roussel
1 INDICATIONS AND USAGE PRIFTIN is a rifamycin antimycobacterial drug indicated in patients 12 years of age and older for the treatment of active pulm…
Xifaxan
rifaximin
Salix Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE To reduce the development of drug-resistant bacteria and maintain the effectiveness of XIFAXAN and other antibacterial drugs, …
Arcalyst
rilonacept
Kiniksa Pharmaceuticals, Ltd.
1 INDICATIONS AND USAGE ARCALYST (rilonacept) is an interleukin-1 blocker indicated for: Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), …
Rilutek
Riluzole
Rhone-Poulenc Rorer Pharmaceuticals, Inc.
Treatment of patients with amyotrophic lateral sclerosis. Riluzole extends survival and/or time to tracheostomy.…
EXSERVAN
riluzole oral film
Aquestive Therapeutics
EXSERVAN is indicated for the treatment of amyotrophic lateral sclerosis (ALS).…
Tiglutik
riluzole oral suspension
Italfarmaco SpA
1 INDICATIONS AND USAGE TIGLUTIK is indicated for the treatment of amyotrophic lateral sclerosis (ALS). TIGLUTIK is indicated for the treatment of amy…
Wayrilz
rilzabrutinib
Genzyme Corporation (A Sanofi Company)
1 INDICATIONS AND USAGE WAYRILZ is indicated for the treatment of adult patients with persistent or chronic immune thrombocytopenia (ITP) who have had…
NURTEC ODT
RIMEGEPANT SULFATE
Pfizer Laboratories Div Pfizer Inc
1 INDICATIONS AND USAGE NURTEC ODT is a calcitonin gene-related peptide receptor antagonist indicated for the: acute treatment of migraine with or wit…
Adempas
riociguat
Bayer
1 INDICATIONS AND USAGE Adempas is a soluble guanylate cyclase (sGC) stimulator indicated for the treatment of adults with: • Persistent/recurrent Chr…
EVRYSDI
RISDIPLAM
Genentech, Inc.
1 INDICATIONS AND USAGE EVRYSDI is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. EVRYSDI is a survival…
Norvir Powder for Oral Suspension
Ritonavir powder for oral suspension
AbbVie Inc.
Used in combination with other antiretroviral agents for the treatment of patients with HIV-1 infection…
Rituxan
rituximab
Genentech, Inc.
1 INDICATIONS AND USAGE RITUXAN HYCELA is a combination of rituximab, a CD20-directed cytolytic antibody, and hyaluronidase human, an endoglycosidase,…
Rituxan SC
rituximab and recombinant human hyaluronidase
Genentech, Inc.
Treatment of adult patients with previously untreated and previously treated CLL in combination with fludarabine and cyclophosphamide ||| Treatment of…
XARELTO
RIVAROXABAN
Janssen Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE XARELTO is a factor Xa inhibitor indicated: to reduce risk of stroke and systemic embolism in nonvalvular atrial fibrillation …
Istodax
romidepsin
Celgene Corporation
1 INDICATIONS AND USAGE ISTODAX is indicated for the treatment of cutaneous T-cell lymphoma (CTCL) in adult patients who have received at least one pr…
Nplate
ROMIPLOSTIM
Amgen, Inc
1 INDICATIONS AND USAGE Nplate is a thrombopoietin receptor agonist indicated for the treatment of thrombocytopenia in: Adult patients with immune thr…
Besremi
ropeginterferon alfa-2b-njft
PharmaEssentia
1 INDICATIONS AND USAGE BESREMi is indicated for the treatment of adults with polycythemia vera. BESREMi is an interferon alfa-2b indicated for the tr…
Crestor
rosuvastatin
iPR Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE CRESTOR is indicated: • To reduce the risk of major adverse cardiovascular (CV) events (CV death, nonfatal myocardial infarcti…
Rystiggo
ROZANOLIXIZUMAB
UCB, Inc.
1 INDICATIONS AND USAGE RYSTIGGO is indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine re…
Rubraca
rucaparib
pharma&
1 INDICATIONS AND USAGE RUBRACA is a poly (ADP-ribose) polymerase (PARP) inhibitor indicated: Ovarian cancer for the maintenance treatment of adult pa…
Banzel
rufinamide
Eisai, Inc.
1 I NDICATIONS AND USAGE BANZEL is indicated for adjunctive treatment of seizures associated with Lennox-Gastaut Syndrome in pediatric patients 1 year…
JAKAFI
RUXOLITINIB
Incyte Corporation
1. INDICATIONS AND USAGE Jakafi is a kinase inhibitor indicated for treatment of: intermediate or high-risk myelofibrosis, including primary myelofibr…
Rydapt
RYDAPT
Novartis Pharmaceuticals Corporation
1 INDICATIONS AND USAGE RYDAPT is a kinase inhibitor indicated for the treatment of adult patients with: Newly diagnosed acute myeloid leukemia (AML) …
Sucraid
Sacrosidase
QOL Medical, LLC
INDICATIONS AND USAGE Sucraid ® (sacrosidase) Oral Solution is indicated for the treatment of sucrase deficiency, which is part of congenital sucrase-…
Kuvan
sapropterin
BioMarin Pharmaceutical, Inc.
1 INDICATIONS AND USAGE KUVAN ® is indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients one month of age and older wit…
Sapropterin Dihydrochloride
SAPROPTERIN DIHYDROCHLORIDE
Aurobindo Pharma Limited
1 INDICATIONS AND USAGE Sapropterin dihydrochloride tablets are indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients o…
Leukine
Sargramostim
Immunex Corporation
1 INDICATIONS AND USAGE LEUKINE is a leukocyte growth factor indicated: To shorten time to neutrophil recovery and to reduce the incidence of severe a…
Enspryng
SATRALIZUMAB
Genentech, Inc.
1 INDICATIONS AND USAGE ENSPRYNG is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquap…
Oncoscint CR/OV
Satumomab pendetide
Cytogen Corporation
For determining the extent and location of extraphepatic malignant disease in patients with known colorectal and ovarian cancer.…