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.
Ryplazim
plasminogen, human-tvmh
ProMetic BioTherapeutics, Inc.
Treatment of patients with plasminogen deficiency type 1 (hypoplasminogenemia).…
Mozobil
plerixafor
Genzyme Corporation
1 INDICATIONS AND USAGE Mozobil is indicated in combination with filgrastim to mobilize hematopoietic stem cells (HSCs) to the peripheral blood for co…
Redemplo
plozasiran
Arrowhead Pharmaceuticals, Inc.
as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS)…
POLIVY
POLATUZUMAB VEDOTIN
Genentech, Inc.
1 INDICATIONS AND USAGE POLIVY is a CD79b-directed antibody and microtubule inhibitor conjugate indicated: in combination with a rituximab product, cy…
Gliadel
polifeprosan 20 with carmustine
Guilford Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE GLIADEL Wafer is indicated for the treatment of patients with: newly-diagnosed high-grade glioma as an adjunct to surgery and …
POMALYST�
pomalidomide
Celgene Corporation
1 INDICATIONS AND USAGE Pomalidomide capsules are a thalidomide analogue indicated for the treatment of adult patients: in combination with dexamethas…
Iclusig
ponatinib
Takeda Pharmaceuticals U.S.A., Inc.
1 INDICATIONS AND USAGE ICLUSIG ® is indicated for the treatment of adult patients with: Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia…
Photofrin
porfimer
Axcan Scandipharm Inc.
1 INDICATIONS AND USAGE PHOTOFRIN is a photoactivated radical generator indicated for: Esophageal Cancer ( 1.1 ) Palliation of patients with completel…
Noxafil
posaconazole
Merck Sharp & Dohme LLC
1 INDICATIONS AND USAGE Noxafil is an azole antifungal indicated as follows: Noxafil is indicated for the treatment of invasive aspergillosis as follo…
Urocit-K
Potassium citrate
University of Texas Health Science Center at Dallas
1 INDICATIONS AND USAGE 1.1 Renal Tubular Acidosis (RTA) with Calcium Stones Potassium citrate is indicated for the management of renal tubular acidos…
ThyroShield
Potassium Iodide Oral Solution
Fleming & Company, Pharmaceuticals
For use as a thyroid blocking agent in pediatric patients exposed to radiactive iodine…
Veopoz
pozelimab-bbfg
Regeneron Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE VEOPOZ is indicated for the treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losi…
Zevaskyn
PRADEMAGENE ZAMIKERACEL
Abeona Therapeutics Inc.
1 INDICATIONS AND USAGE ZEVASKYN is indicated for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bull…
Folotyn
pralatrexate
Acrotech Biopharma LLC
1 INDICATIONS AND USAGE FOLOTYN is indicated for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). This indicat…
Protopam Chloride
PRALIDOXIME CHLORIDE
Baxter Healthcare Corporation
INDICATIONS AND USAGE PROTOPAM Chloride is indicated as an antidote: 1. In the treatment of poisoning due to those pesticides and chemicals (e.g., ner…
GAVRETO�
pralsetinib
Rigel Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE GAVRETO is a kinase inhibitor indicated for treatment of: Adult patients with metastatic rearranged during transfection (RET )…
Prednisolone
PREDNISOLONE
Aurobindo Pharma Limited
INDICATIONS AND USAGE 1. Endocrine disorders . Primary or secondary adrenocortical insufficiency (hydrocortisone or cortisone is the first choice; syn…
Prednisolone Sodium Phosphate
PREDNISOLONE SODIUM PHOSPHATE
Proficient Rx LP
INDICATIONS AND USAGE Prednisolone Sodium Phosphate Oral Solution (15 mg Prednisolone per 5 mL) is indicated in the following conditions: 1. Allergic …
Prednisone
PREDNISONE
A-S Medication Solutions
INDICATIONS Prednisone tablets are indicated in the following conditions: 1. Endocrine Disorders Primary or secondary adrenocortical insufficiency (hy…
Pretomanid
Pretomanid
Global Alliance for TB Drug Development
1 INDICATIONS AND USAGE Limited Population: Pretomanid Tablet is indicated, as part of a combination regimen with bedaquiline and linezolid for the tr…
HEMANGEOL
propranolol
Eton Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE HEMANGEOL oral solution contains the beta-adrenergic blocker propranolol hydrochloride and is indicated for the treatment of p…
Ceprotin
Protein C concentrate
Baxalta US, Inc.
Prevention and treatment of venous thrombosis and purpura fulminans…
Kcentra
prothrombin complex concentrate (human)
CSL Behring
Urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist (VKA, e.g., warfarin) therapy in adult patients with acute m…
Balfaxar
prothrombin complex concentrate, human-lans
Octapharma USA, Inc.
urgent reversal of acquired coagulation factor deficiency induced by Vitamin K antagonist (VKA, e.g., warfarin) therapy in adult patients with need fo…
Curosurf
Pulmonary surfactant replacement, porcine
Chiesi USA, Inc.
1 INDICATIONS AND USAGE CUROSURF ® (poractant alfa) Intratracheal Suspension is indicated for the rescue treatment of Respiratory Distress Syndrome (R…
Pyridostigmine Bromide
PYRIDOSTIGMINE BROMIDE
ANI Pharmaceuticals, Inc.
INDICATIONS AND USAGE Pyridostigmine Bromide Tablets USP are useful in the treatment of myasthenia gravis.…
Pyridostigmine
PYRIDOSTIGMINE BROMIDE ORAL
Novitium Pharma LLC
INDICATIONS & USAGE Pyridostigmine Bromide Oral Solution, USP is useful in the treatment of myasthenia gravis.…
Vanflyta
quizartinib
Daiichi Sankyo, Inc.
1 INDICATIONS AND USAGE VANFLYTA is indicated in combination with standard cytarabine and anthracycline induction and cytarabine consolidation, and as…