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.
Kymriah (tisagenlecleucel)
(tisagenlecleucel) Autologous T Cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19
Novartis Pharmaceuticals Corporation
Treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse…
Gleolan
5-aminolevulinic acid
NX Development Corporation
1 INDICATIONS AND USAGE Gleolan is indicated in patients with glioma [suspected World Health Organization (WHO) Grades III or IV on preoperative imagi…
Orencia
abatacept
Bristol-Myers Squibb Co.
1 INDICATIONS AND USAGE ORENCIA is a selective T cell costimulation modulator indicated for: • the treatment of adult patients with moderately to seve…
CALQUENCE
acalabrutinib
Acerta Pharma, LLC (a member of the AstraZeneca Group)
1 INDICATIONS AND USAGE CALQUENCE is a kinase inhibitor indicated: • In combination with bendamustine and rituximab for the treatment of adult patient…
Acetylcysteine
ACETYLCYSTEINE
Hospira, Inc.
INDICATIONS AND USAGE Acetylcysteine is indicated as adjuvant therapy for patients with abnormal, viscid, or inspissated mucous secretions in such con…
Cetylev
acetylcysteine effervescent tablets for oral solution
Arbor Pharmaceuticals, Inc.
Indicated to prevent or lessen hepatic injury after ingestion of a potentially hepatotoxic quantity of acetaminophen in patients with acute ingestion …
Attruby
ACORAMIDIS HYDROCHLORIDE
BridgeBio Pharma, Inc.
1 INDICATIONS AND USAGE ATTRUBY is indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-…
Avaclyr
acyclovir
Fera Pharmaceuticals
1 INDICATIONS AND USAGE AVACLYR is a sterile topical antiviral indicated for the treatment of acute herpetic keratitis (dendritic ulcers) in patients …
KRAZATI
ADAGRASIB
Mirati Therapeutics, Inc
1 INDICATIONS AND USAGE KRAZATI is an inhibitor of the RAS GTPase family indicated for: Non-small cell lung cancer (NSCLC)* • As a single agent, for t…
HUMIRA
adalimumab
AbbVie, Inc.
1 INDICATIONS AND USAGE HUMIRA is a tumor necrosis factor (TNF) blocker indicated for: Reducing signs and symptoms, inducing major clinical response, …
SCENESSE
afamelanotide
Clinuvel Inc.
SCENESSE ® is indicated to increase pain free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoporphyr…
Tecelra
afamitresgene autoleucel
USWM CT, LLC
1 INDICATIONS AND USAGE TECELRA is a melanoma-associated antigen A4-(MAGE-A4)-directed genetically modified autologous T cell immunotherapy indicated …
Gilotrif
afatinib
Boehringer Ingelheim Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE GILOTRIF is a kinase inhibitor indicated for: First-line treatment of patients with metastatic non-small cell lung cancer (NSC…
Myqorzo
aficamten
Cytokinetics Inc.
1 INDICATIONS AND USAGE MYQORZO is indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve fu…
Eylea
aflibercept
Regeneron Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE EYLEA HD is indicated for the treatment of: EYLEA HD is a vascular endothelial growth factor (VEGF) inhibitor indicated for th…
Fabrazyme
AGALSIDASE BETA
Genzyme Corporation
1 INDICATIONS AND USAGE FABRAZYME ® is indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disea…
Albenza
Albendazole
Impax Laboratories, LLC
1 INDICATIONS AND USAGE ALBENZA is an anthelmintic drug indicated for: Treatment of parenchymal neurocysticercosis due to active lesions caused by lar…
Proleukin
Aldesleukin
Chiron Corporation
1 INDICATIONS AND USAGE Proleukin is a lymphocyte growth factor indicated for: The treatment of adults with metastatic renal cell carcinoma. ( 1.1 ) T…
Alecensa
alectinib
Hoffmann-La Roche, Inc. (c/o Genentech, Inc.)
1 INDICATIONS AND USAGE ALECENSA is a kinase inhibitor indicated for: adjuvant treatment in adult patients following tumor resection of anaplastic lym…
Campath
alemtuzumab
Genzyme Corporation
1 INDICATIONS AND USAGE CAMPATH is indicated as a single agent for the treatment of B-cell chronic lymphocytic leukemia (B-CLL). CAMPATH is a CD52-dir…
Ceredase
Alglucerase injection
Genzyme Corporation
Long-term enzyme replacement therapy for children, adolescents and adult patients with a confirmed diagnosis of Type I Gaucher disease who exhibit sig…
Lumizyme
ALGLUCOSIDASE ALFA
Genzyme Corporation
1 INDICATIONS AND USAGE LUMIZYME ® is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α-glucosidase [G…
Praluent
alirocumab
Regeneron Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE PRALUENT ® is indicated: To reduce the risk of major adverse cardiovascular (CV) events (coronary heart disease death, myocard…
Panretin
Alitretinoin
Advanz Pharma (US) Corp.
1 INDICATIONS AND USAGE 1.1 Kaposi’s Sarcoma PANRETIN GEL is indicated for topical treatment of cutaneous lesions in adults with AIDS related Kaposi’s…
StrataGraft
allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen-dsat
Stratatech Corporation, a Mallinckrodt company
Treatment of adults with thermal burns containing intact dermal elements for which surgical intervention is clinically indicated (deep partial-thickne…
Rethymic
allogeneic processed thymus tissue-agdc
Enzyvant Therapeutics GmbH
1 INDICATIONS AND USAGE RETHYMIC ® is indicated for immune reconstitution in pediatric patients with congenital athymia. RETHYMIC is indicated for imm…
Aloprim for Injection
Allopurinol sodium
Catalytica Pharmaceuticals, Inc
1 INDICATIONS AND USAGE Allopurinol Sodium for Injection is indicated for the management of adult and pediatric patients with leukemia, lymphoma, and …
Vijoice
alpelisib
Novartis Pharmaceuticals Corporation
1 INDICATIONS AND USAGE VIJOICE is indicated for the treatment of adult and pediatric patients 2 years of age and older with severe manifestations of …