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.
Camzyos
mavacamten
MyoKardia, Inc. (A Wholly-Owned Subsidiary of Bristol Myers Squibb)
1 INDICATIONS AND USAGE CAMZYOS ® is indicated for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive…
Xolremdi
mavorixafor
X4 Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE XOLREMDI is indicated in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and m…
VERMOX 500 mg chewable tablets
mebendazole
Janssen Pharmaceutical Research & Development, LLC
1 INDICATIONS AND USAGE VERMOX™ CHEWABLE is indicated for the treatment of patients one year of age and older with gastrointestinal infections caused …
Increlex
Mecasermin
Eton Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE INCRELEX (mecasermin) injection is indicated for the treatment of growth failure in pediatric patients 2 years of age and olde…
Iplex
mecasermin rinfabate
Insmed, Inc.
Treatment of growth failure in children with severe primary IGF-1 deficiency (Primary IGFD) or with growth hormone (GH) gene deletion who have develop…
Valchlor
meclorethamine
Helsinn Birex Pharmaceuticals Ltd.
1 INDICATIONS AND USAGE VALCHLOR is indicated for the topical treatment of Stage IA and IB mycosis fungoides-type cutaneous T-cell lymphoma in patient…
Lariam
mefloquine HCL
Hoffmann-La Roche, Inc.
Treatment of acute malaria due to Plasmodium falciparum and Plasmodium vivax ||| Prophylaxis of Plasmodium falciparum malaria which is resistant to ot…
Megace
Megestrol acetate
Bristol-Myers Squibb Pharmaceutical Research Institute
INDICATIONS AND USAGE Megestrol acetate oral suspension, USP is indicated for the treatment of anorexia, cachexia, or an unexplained, significant weig…
Mobic
meloxicam
Avondale Pharmaceuticals
1 INDICATIONS AND USAGE Meloxicam tablet USP is a non-steroidal anti-inflammatory drug indicated for: Osteoarthritis (OA) ( 1.1 ) Rheumatoid Arthritis…
Alkeran For Injection
Melphalan
Glaxo Wellcome Inc.
INDICATIONS AND USAGE Melphalan Hydrochloride for Injection is indicated for the palliative treatment of patients with multiple myeloma for whom oral …
Pepaxto
melphalan flufenamide
Oncopeptides AB
Treatment, in combination with dexamethasone, of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lin…
NUCALA
mepolizumab
GlaxoSmithKline LLC
1 INDICATIONS AND USAGE NUCALA is an interleukin-5 (IL-5) antagonist monoclonal antibody (IgG1 kappa) indicated for: • Add-on maintenance treatment of…
Purixan
mercaptopurine oral solution
Nova Laboratories Limited
1 INDICATIONS AND USAGE PURIXAN is a nucleoside metabolic inhibitor indicated for the treatment of patients with acute lymphoblastic leukemia (ALL) as…
Lialda
mesalamine; 5-aminosalicylic acid
Takeda Development Center Americas, Inc.
1 INDICATIONS AND USAGE LIALDA ® is indicated for the: induction and maintenance of remission in adult patients with mildly to moderately active ulcer…
Mesnex
Mesna
Degussa Corporation
1 INDICATIONS AND USAGE MESNEX is indicated as a prophylactic agent in reducing the incidence of ifosfamide-induced hemorrhagic cystitis. Limitation o…
Xatmep
methotrexate oral solution
Silvergate Pharmaeuticals, Inc.
1 INDICATIONS AND USAGE XATMEP is a folate analog metabolic inhibitor indicated for the: Treatment of pediatric patients with acute lymphoblastic leuk…
Methotrexate
Methotrexate sodium
Lederle Laboratories
1 INDICATIONS AND USAGE Methotrexate tablets are a diydrofolate reductase inhibitor indicated for the: • Treatment of adults and pediatric patients wi…
ProvayBlue
methylene blue0.5%
Provepharm SAS
1 INDICATIONS AND USAGE PROVAYBLUE is indicated for the treatment of pediatric and adult patients with acquired methemoglobinemia. PROVAYBLUE (methyle…
METHYLPHENIDATE HYDROCHLORIDE
METHYLPHENIDATE HYDROCHLORIDE
Granules Pharmaceuticals Inc.
1 INDICATIONS AND USAGE Methylphenidate hydrochloride extended-release capsules are indicated for the treatment of Attention Deficit Hyperactivity Dis…
Methylprednisolone
METHYLPREDNISOLONE
Bryant Ranch Prepack
INDICATIONS AND USAGE Methylprednisolone tablets are indicated in the following conditions: 1. Endocrine Disorders Primary or secondary adrenocortical…
Methylprednisolone Acetate
METHYLPREDNISOLONE ACETATE
A-S Medication Solutions
INDICATIONS AND USAGE A. For Intramuscular Administration When oral therapy is not feasible and the strength, dosage form, and route of administration…
Dyural 80-Lm
METHYLPREDNISOLONE ACETATE, LIDOCAINE HYDROCHLORIDE, BUPIVACAINE HYDROCHLORIDE, POVIDINE IODINE, ISOPROPYL ALCOHOL
Advanced Rx Pharmacy of Tennessee, LLC
1 INDICATIONS AND USAGE Bupivacaine Hydrochloride Injection is indicated in adults for the production of local or regional anesthesia or analgesia for…
Dyural 40 Kit
METHYLPREDNISOLONE ACETATE, LIDOCAINE HYDROCHLORIDE, BUPIVACAINE HYDROCHLORIDE, POVIDINE IODINE, SODIUM CHLORIDE, ISOPROPYL ALCOHOL
Advanced Rx Pharmacy of Tennessee, LLC
1 INDICATIONS AND USAGE Bupivacaine Hydrochloride Injection is indicated in adults for the production of local or regional anesthesia or analgesia for…
Methylprednisolone Sodium Succinate
METHYLPREDNISOLONE SODIUM SUCCINATE
Hikma Pharmaceuticals USA Inc.
INDICATIONS AND USAGE When oral therapy is not feasible, and the strength, dosage form, and route of administration of the drug reasonably lend the pr…
MYALEPT
metreleptin
Chiesi Farmaceutici S.p.A.
1 INDICATIONS AND USAGE MYALEPT is a leptin analog indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficie…
Metrogel
Metronidazole (topical)
Galderma Laboratories, Inc.
1 INDICATIONS AND USAGE METROGEL, 1% is indicated for the topical treatment of inflammatory lesions of rosacea. METROGEL, 1% is a nitroimidazole indic…
NAYZILAM
midazolam
UCB, Inc.
1 INDICATIONS AND USAGE NAYZILAM is indicated for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizur…