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.
Votrient
pazopanib
Novartis Pharmaceuticals Corp
1 INDICATIONS AND USAGE VOTRIENT is a kinase inhibitor indicated for the treatment of adults with: advanced renal cell carcinoma (RCC). ( 1.1 ) advanc…
Oncaspar; Asparlas
Pegaspargase
Servier Pharmaceuticals LLC
1 INDICATIONS AND USAGE ONCASPAR is an asparagine specific enzyme indicated as a component of a multi-agent chemotherapeutic regimen for treatment of …
Empaveli
PEGCETACOPLAN
Apellis Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE EMPAVELI is a complement inhibitor indicated: for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PN…
Neulasta
Pegfilgrastim
Amgen, Inc.
1 INDICATIONS AND USAGE ZIEXTENZO is a leukocyte growth factor indicated to • Decrease the incidence of infection, as manifested by febrile neutropeni…
Sylatron
peginterferon alfa-2b
Merck, Sharpe & Dohme Corp.
Adjuvant treatment of melanoma with microscopic or gross nodal involvement within 84 days of definitive surgical resection including complete lymphade…
Krystexxa
pegloticase
Horizon Therapeutics Ireland DAC
1 INDICATIONS AND USAGE KRYSTEXXA ® (pegloticase) is indicated, for the treatment of chronic gout in adult patients refractory to conventional therapy…
ELFABRIO
PEGUNIGALSIDASE ALFA
Chiesi USA, Inc.
1 INDICATIONS AND USAGE ELFABRIO is indicated for the treatment of adults with confirmed Fabry disease. ELFABRIO is a hydrolytic lysosomal neutral gly…
Palynziq
PEGVALIASE-PQPZ
BioMarin Pharmaceutical Inc.
1 INDICATIONS AND USAGE PALYNZIQ is indicated to reduce blood phenylalanine concentrations in adult and pediatric patients 12 years of age and older w…
Somavert
Pegvisomant
Sensus Corporation
1 INDICATIONS AND USAGE SOMAVERT is indicated for the treatment of acromegaly in patients who have had an inadequate response to surgery or radiation …
Loargys
pegzilarginase-nbln
Immedica Pharma AB
1 INDICATIONS AND USAGE LOARGYS is indicated for the treatment of hyperargininemia in adult and pediatric patients 2 years of age and older with Argin…
KEYTRUDA
pembrolizumab
MSD International Business GmbH
1 INDICATIONS AND USAGE KEYTRUDA QLEX is a combination of pembrolizumab, a programmed death receptor-1 (PD-1)-blocking antibody, and berahyaluronidase…
Alimta
pemetrexed disodium
Eli Lilly and Company
1 INDICATIONS AND USAGE ALIMTA ® is a folate analog metabolic inhibitor indicated: in combination with pembrolizumab and platinum chemotherapy, for th…
PEMAZYRE
Pemigatinib
Incyte Corporation
1 INDICATIONS AND USAGE PEMAZYRE is a kinase inhibitor indicated: for the treatment of adults with previously treated, unresectable locally advanced o…
Penicillamine
PENICILLAMINE
Endo USA, Inc.
INDICATIONS Penicillamine capsules are indicated in the treatment of Wilson's disease, cystinuria, and in patients with severe, active rheumatoid arth…
Nebupent
Pentamidine isethionate
Fujisawa USA, Inc.
INDICATIONS AND USAGE: NebuPent is indicated for the prevention of Pneumocystis jiroveci pneumonia (PJP) in high-risk, HIV-infected patients defined b…
Elmiron
Pentosan polysulfate sodium
Alza Corporation
INDICATIONS AND USAGE ELMIRON ® (pentosan polysulfate sodium) is indicated for the relief of bladder pain or discomfort associated with interstitial c…
Nipent
pentostatin for injection
SuperGen, Inc.
INDICATIONS AND USAGE NIPENT is indicated as single-agent treatment for both untreated and alpha-interferon-refractory hairy cell leukemia patients wi…
TURALIO
pexidartinib
Daiichi Sankyo, Inc.
1 INDICATIONS AND USAGE TURALIO is indicated for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with…
Sezaby
phenobarbital sodium
Sun Pharmaceutical Industries, Inc.
1 INDICATIONS AND USAGE SEZABY is indicated for the treatment of neonatal seizures in term and preterm infants. SEZABY is a barbiturate indicated for …
PHYTONADIONE PHYTONADIONE
PHYTONADIONE
Dr.Reddy's Laboratories Inc
INDICATIONS AND USAGE Phytonadione injectable emulsion, USP is indicated in the following coagulation disorders which are due to faulty formation of f…
Salagen
Pilocarpine
MGI Pharma, Inc.
INDICATIONS & USAGE SALAGEN ® Tablets are indicated for 1) the treatment of symptoms of dry mouth from salivary gland hypofunction caused by radiother…
Pirfenidone
PIRFENIDONE
AvKARE
1 INDICATIONS AND USAGE Pirfenidone capsules are indicated for the treatment of idiopathic pulmonary fibrosis (IPF). Pirfenidone is a pyridone indicat…
Pirfenidone Capsule, 267 Mg
PIRFENIDONE CAPSULE, 267 MG
Macleods Pharmaceuticals Limited
1 INDICATIONS AND USAGE Pirfenidone capsules are indicated for the treatment of idiopathic pulmonary fibrosis (IPF). Pirfenidone capsules are a pyrido…
PIROXICAM
PIROXICAM
Unichem Pharmaceuticals (USA), Inc.
1 INDICATIONS AND USAGE Piroxicam capsules are indicated: • For relief of the signs and symptoms of osteoarthritis. • For relief of the signs and symp…
Jaypirca
pirtobrutinib
Loxo Oncology, Inc.
1 INDICATIONS AND USAGE JAYPIRCA ® is a kinase inhibitor indicated for the treatment of: Adult patients with relapsed or refractory mantle cell lympho…
WAKIX
Pitolisant
Harmony Biosciences Management, Inc.
1 INDICATIONS AND USAGE WAKIX is indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy in patients 6 years of age and older w…