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.
Valproic Acid
VALPROIC ACID
Direct_Rx
These highlights do not include all the information needed to use VALPROIC ACID CAPSULES safely and effectively. See full prescribing information for …
Valstar
Valrubicin
Anthra Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE VALSTAR is an anthracycline topoisomerase inhibitor indicated for intravesical therapy of BCG-refractory carcinoma in situ (CI…
AGAMREE
VAMOROLONE
Catalyst Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE AGAMREE is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. AGAMREE is a…
CAPRELSA(r)
vandetanib
Genzyme Corporation
1 INDICATIONS AND USAGE CAPRELSA is indicated for the treatment of symptomatic or progressive medullary thyroid cancer in patients with unresectable l…
ALYFTREK
VANZACAFTOR, TEZACAFTOR, AND DEUTIVACAFTOR
Vertex Pharmaceuticals Incorporated
1 INDICATIONS AND USAGE ALYFTREK is indicated for the treatment of cystic fibrosis (CF) in patients 6 years of age and older who have at least one F50…
ALYFTREK
VANZACAFTOR, TEZACAFTOR, AND DEUTIVACAFTOR
Vertex Pharmaceuticals Incorporated
1 INDICATIONS AND USAGE ALYFTREK is indicated for the treatment of cystic fibrosis (CF) in patients 6 years of age and older who have at least one F50…
VARIZIG
Varicella Zoster Immune Globulin (Human)
Cangene bioPharma, Inc.
Post exposure prophylaxis of varicella in high risk individuals to reduce the severity of varicella…
VPRIV
VELAGLUCERASE ALFA
Takeda Pharmaceuticals America, Inc.
1 INDICATIONS AND USAGE VPRIV is indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease. VPRIV is a hydroly…
Lamzede
VELMANASE ALFA-TYCV
Chiesi USA, Inc.
1 INDICA TIONS AND USAGE LAMZEDE is indicated for the treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediat…
Zelboraf
VEMURAFENIB
Genentech, Inc.
1 INDICATIONS AND USAGE ZELBORAF ® is a kinase inhibitor indicated for the treatment of patients with unresectable or metastatic melanoma with BRAF V6…
VENCLEXTA
venetoclax
AbbVie, Inc
1 INDICATIONS AND USAGE VENCLEXTA is a BCL-2 inhibitor indicated: For the treatment of adult patients with chronic lymphocytic leukemia (CLL) or small…
MEPSEVII
VESTRONIDASE ALFA
Ultragenyx Pharmaceutical Inc.
1 INDICATIONS AND USAGE MEPSEVII is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome). …
VIGABATRIN
VIGABATRIN
Hikma Pharmaceuticals USA Inc
1 INDICATIONS AND USAGE Vigabatrin tablets are indicated for the treatment of: Refractory Complex Partial Seizures as adjunctive therapy in patients 2…
Viltepso
VILTOLARSEN
NS Pharma, Inc.
1 INDICATIONS AND USAGE VILTEPSO is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the …
Marqibo
vinCRIStine sulfate LIPOSOME injection
Acrotech Biopharma LLC
Treatment of adult patients with Philadelphia chromosome-negative (Ph-) acute lymphoblastic leukemia (ALL) in second or greater relapse or whose disea…
Vonvendi
von Willebrand factor (recombinant)
Takeda Pharmaceuticals U.S.A., Inc.
For use in adults (age 18 and older) diagnosed with von Willebrand disease (VWD) for routine prophylaxis to reduce the frequency of bleeding episodes …
Wilate
von Willebrand Factor/Coagulation Factor VIII Complex (Human)
Octapharma USA, Inc.
Treatment of spontaneous or trauma-induced bleeding episodes in patients with severe von Willebrand disease (VWD) as well as patients with mild or mod…
Voranigo
vorasidenib
Servier Pharmaceuticals LLC
1 INDICATIONS AND USAGE VORANIGO is indicated for the treatment of adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligode…
LUXTURNA
voretigene neparvovec-rzyl
Spark Therapeutics, Inc.
1 INDICATIONS AND USAGE LUXTURNA (voretigene neparvovec-rzyl) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pa…
Zolinza
vorinostat
Merck & Co., Inc.
1 INDICATIONS AND USAGE ZOLINZA ® is indicated for the treatment of cutaneous manifestations in patients with cutaneous T-cell lymphoma who have progr…
Voxzogo 0.4Mg
VOSORITIDE
BioMarin Pharmaceutical Inc.
1 INDICATIONS AND USAGE VOXZOGO is indicated to increase linear growth in pediatric patients with achondroplasia with open epiphyses. This indication …
OXBRYTA
VOXELOTOR
Global Blood Therapeutics, Inc, A subsidiary of Pfizer Inc.
1 INDICATIONS AND USAGE OXBRYTA is indicated for the treatment of sickle cell disease (SCD) in adults and pediatric patients 4 years of age and older.…
AMVUTTRA
VUTRISIRAN
Alnylam Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE AMVUTTRA is a transthyretin-directed small interfering RNA indicated for the treatment of: the polyneuropathy of hereditary tr…
Vyjuvek
VYJUVEK
Krystal Biotech, Inc.
1 INDICATIONS AND USAGE VYJUVEK is indicated for the treatment of wounds in adult and pediatric patients with dystrophic epidermolysis bullosa (DEB) w…
Sterile Diluent For Treprostinil
WATER
Dr.Reddy's Laboratories Inc.,
1 INDICATIONS AND USAGE Treprostinil injection is a prostacyclin vasodilator indicated for: Treatment of pulmonary arterial hypertension (PAH; WHO Gro…
Hivid
Zalcitabine
Hoffmann-La Roche, Inc.
Combination therapy with zidovudine is indicated for the treatment of adult patients with advanced HIV infection (CD4 count < 300) who have demonstrat…
Ziihera
zanidatamab-hrii
Jazz Pharmaceuticals Ireland Limited
1 INDICATIONS AND USAGE ZIIHERA is indicated for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) bi…
BRUKINSA
zanubrutinib
BeOne Medicines USA, Inc.
1 INDICATIONS AND USAGE BRUKINSA is a kinase inhibitor indicated for the treatment of adult patients with: Mantle cell lymphoma (MCL) who have receive…