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.
Raloxifene Hydrochloride
RALOXIFENE
Aphena Pharma Solutions - Tennessee, LLC
1 INDICATIONS AND USAGE Raloxifene hydrochloride tablet, USP is an estrogen agonist/ antagonist indicated for: Treatment and prevention of osteoporosi…
Raloxifene Hydrochloride
RALOXIFENE HYDROCHLORIDE
NorthStar Rx LLC
1 INDICATIONS AND USAGE Raloxifene hydrochloride is an estrogen agonist/antagonist indicated for: Treatment and prevention of osteoporosis in postmeno…
CYRAMZA
ramucirumab
Eli Lilly and Company
1 INDICATIONS AND USAGE CYRAMZA ® is a human vascular endothelial growth factor receptor 2 (VEGFR2) antagonist indicated: as a single agent or in comb…
Ranitidine
RANITIDINE
Rising Pharma Holdings, Inc.
Use(s) relieves heartburn associated with acid indigestion and sour stomach prevents heartburn associated with acid indigestion and sour stomach broug…
Elitek
rasburicase
Sanofi-Synthelabo Research
1 INDICATIONS AND USAGE Elitek is indicated for the initial management of plasma uric acid levels in pediatric and adult patients with leukemia, lymph…
Ultomiris
RAVULIZUMAB
Alexion Pharmaceuticals Inc.
1 INDICATIONS AND USAGE ULTOMIRIS is a complement inhibitor indicated for: the treatment of adult and pediatric patients one month of age and older wi…
ABthraxTM
raxibacumab
Human Genome Sciences, Inc.
1 INDICATIONS AND USAGE Raxibacumab is indicated for the treatment of adult and pediatric patients with inhalational anthrax due to Bacillus anthracis…
Idelvion
recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP)
CSL Behring, LLC
Indicated in children and adults with hemophilia B (congenital Factor IX deficiency) for the (1) on-demand control and prevention of bleeding episodes…
Myozyme
Recombinant human acid alpha-glucosidase; alglucosidase alfa
Genzyme Corporation
Myozyme for use in patients with Pompe disease (GAA deficiency). Alglucosidase alfa has been shown to improve ventilator-free survival in patients wit…
ATryn
recombinant human antithrombin
GTC Biotherapeutics, Inc.
Prevention of peri-operative and peri-partum thromboembolic events, in hereditary antithrombin deficient patients.…
Luveris
Recombinant human luteinizing hormone
EMD Serono, Inc.
Luveris (lutropin alfa for injection), concomitantly administered with Gonal-f (follitropin alfa for injection), is indicated for stimulation of folli…
Stivarga
regorafenib
Bayer HealthCare Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE STIVARGA is a kinase inhibitor indicated for the treatment of adult patients with: • Metastatic colorectal cancer (CRC) who ha…
Ryoncil
remestemcel-L-rknd
Mesoblast, Inc.
1 INDICATIONS AND USAGE RYONCIL is indicated for the treatment of steroid refractory acute graft versus host disease (SR-aGvHD) in pediatric patients …
H.P. Acthar Gel
repository corticotropin or adrenocorticotropic hormone
Questcor Pharmaceuticals, Inc.
To treat infantile spasms…
Augtyro
repotrectinib
Bristol Myers Squibb Company
1 INDICATIONS AND USAGE AUGTYRO is a kinase inhibitor indicated for the treatment of • adult patients with locally advanced or metastatic ROS1- positi…
Respigam
Respiratory syncytial virus immune globulin (Human)
MedImmune & Massachussetts Public Health Biologics Labs.
For the prevention of serious lower respiratory tract infections caused by RSV in children less than 24 months of age with bronchopulmonary dysplasia …
Zynyz
retifanlimab-dlwr
Incyte Corporation
1 INDICATIONS AND USAGE ZYNYZ is a programmed death receptor-1 (PD-1)–blocking antibody indicated: Squamous Cell Carcinoma of the Anal Canal (SCAC) in…
Encelto
revakinagene taroretcel-lwey
Neurotech Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE ENCELTO is indicated for the treatment of adults with idiopathic macular telangiectasia type 2 (MacTel). ENCELTO is an allogen…
Revuforj
revumenib
Syndax Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE REVUFORJ is a menin inhibitor indicated for: the treatment of relapsed or refractory acute leukemia with a lysine methyltransf…
Rezzayo
rezafungin
Mundipharma GmbH
1 INDICATIONS AND USAGE REZZAYO is an echinocandin antifungal indicated in patients 18 years of age or older who have limited or no alternative option…
WinRho SD
Rho (D) immune globulin intravenous (human)
Rh Pharmaceuticals, Inc.
Treatment of adults and children with chronic and acute immune thrombocytopenic purpura.…
REBETOL
ribavirin
Schering Corporation
INDICATIONS AND USAGE Ribavirin for inhalation solution, USP is indicated for the treatment of hospitalized infants and young children with severe low…
Epioxa HD and Epioxa
riboflavin 5'-phosphate
Glaukos Corporation
1 INDICATIONS AND USAGE EPIOXA HD and EPIOXA are indicated in epithelium-on corneal collagen cross-linking for the treatment of keratoconus in adults …
Photrexa Viscous
riboflavin ophthalmic solution & ultraviolet A
Glaukos Corporation
Indicated for use in corneal collagen crosslinking in combination with the KXL� System for the treatment of progressive keratoconus ||| Treatment of…
Mycobutin
Rifabutin
Adria Laboratories, Inc.
INDICATIONS AND USAGE Rifabutin capsules are indicated for the prevention of disseminated Mycobacterium avium complex (MAC) disease in patients with a…
Rifadin I.V.
Rifampin
Hoechst Marion Roussel
INDICATIONS AND USAGE In the treatment of both tuberculosis and the meningococcal carrier state, the small number of resistant cells present within la…
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…