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.
KETALAR
KETAMINE HYDROCHLORIDE
Par Health USA, LLC
1 INDICATIONS AND USAGE KETALAR (ketamine hydrochloride) injection is indicated: as the sole anesthetic agent for diagnostic and surgical procedures t…
L-Glutamine
L-GLUTAMINE
ANI Pharmaceuticals, Inc.
INDICATIONS For the temporary relief of mild abdominal pain or discomfort, diarrhea, minor joint pain, general weakness, muscle fatigue, sadness, or a…
Lamictal
Lamotrigine
Glaxo Wellcome Research and Development
1 INDICATIONS AND USAGE LAMICTAL XR is indicated for: • adjunctive therapy for primary generalized tonic-clonic seizures (PGTC) and partial-onset seiz…
TAKHZYRO
LANADELUMAB-FLYO
Takeda Pharmaceuticals America, Inc.
1 INDICATIONS AND USAGE TAKHZYRO ® is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients aged…
Somatuline Depot
Lanreotide
IPSEN, Inc.
1 INDICATIONS AND USAGE SOMATULINE DEPOT is a somatostatin analog indicated for: the long-term treatment of acromegalic patients who have had an inade…
ALDURAZYME
LARONIDASE
Genzyme Corporation
1 INDICATIONS AND USAGE ALDURAZYME ® is indicated for the treatment of: adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysa…
VITRAKVI
larotrectinib
Bayer HealthCare Pharmaceuticals Inc.
1 INDICATIONS AND USAGE VITRAKVI is indicated for the treatment of adult and pediatric patients with solid tumors that: have a neurotrophic receptor t…
Harvoni
ledipasvir/sofosbuvir
Gilead Sciences, Inc.
1 INDICATIONS AND USAGE HARVONI is indicated for the treatment of adults and pediatric patients 3 years of age and older with chronic hepatitis C viru…
Lenalidomide
LENALIDOMIDE
Teva Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE Lenalidomide is a thalidomide analogue indicated for the treatment of adult patients with: Multiple myeloma (MM), in combinati…
Joenja
leniolisib
Pharming Technologies B.V.
1 INDICATIONS AND USAGE JOENJA is indicated for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediat…
LENVIMA
lenvatinib
Eisai Inc.
1 INDICATIONS AND USAGE LENVIMA is a kinase inhibitor that is indicated: Differentiated Thyroid Cancer (DTC) For the treatment of adult patients with …
Refluden
Lepirudin
Hoechst Marion Roussel
For anticoagulation in patients with heparin-induced thrombocytopenia and associated thromboembolic disease in order to prevent further thromboembolic…
PREVYMIS
letermovir
Merck Sharp & Dohme LLC
1 INDICATIONS AND USAGE PREVYMIS is a CMV DNA terminase complex inhibitor indicated for: Prophylaxis of cytomegalovirus (CMV) infection and disease in…
Leucovorin calcium
Leucovorin
Immunex Corporation
INDICATIONS AND USAGE Leucovorin calcium rescue is indicated after high dose methotrexate therapy in osteosarcoma. Leucovorin calcium is also indicate…
Lupron Depot-Ped
Leuprolide acetate
AbbVie Endocrine Inc.
1 INDICATIONS AND USAGE LUPRON DEPOT-PED is indicated for the treatment of pediatric patients with central precocious puberty (CPP). LUPRON DEPOT-PED …
AQNEURSA
LEVACETYLLEUCINE
IntraBio Inc
1 INDICATIONS AND USAGE AQNEURSA™ is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and ped…
Carnitor
Levocarnitine
Leadiant Biosciences, Inc.
INDICATIONS AND USAGE CARNITOR ® (levocarnitine) is indicated in the treatment of primary systemic carnitine deficiency. In the reported cases, the cl…
Recorlev
levoketoconazole
Strongbridge Dublin Limited
1 INDICATIONS AND USAGE RECORLEV is indicated for the treatment of endogenous hypercortisolemia in adult patients with Cushing’s syndrome for whom sur…
Fusilev
levoleucovorin
Acrotech Biopharma LLC
1 INDICATIONS AND USAGE Levoleucovorin Injection is indicated for: rescue after high-dose methotrexate therapy in adult and pediatric patients with os…
Orlaam
Levomethadyl acetate hydrochloride
Biodevelopment Corporation
For the management of opiate dependence.…
Physician Ez Use Joint Tunnel And Trigger Kit Ii
LIDOCAINE HYDROCHLORIDE, TRIAMCINOLONE ACETONIDE, POVIDONE-IODINE
Asclemed USA, Inc.
INDICATIONS AND USAGE Intramuscular Where oral therapy is not feasible, injectable corticosteroid therapy, including triamcinolone acetonide injectabl…
Lidoderm Patch
Lidocaine patch 5%
Teikoku Pharma USA, Inc.
INDICATION AND USAGE LIDODERM is indicated for relief of pain associated with post-herpetic neuralgia. It should be applied only to intact skin .…
Lidolog Kit
LIDOCAINE, KENALOG, POVIDONE IODINE
Asclemed USA, Inc.
INDICATIONS AND USAGE Lidocaine hydrochloride injection is indicated for production of local or regional anesthesia by infiltration techniques such as…
Amtagvi
lifileucel
Iovance Biotherapeutics, Inc.
1 INDICATIONS AND USAGE AMTAGVI is a tumor-derived autologous T cell immunotherapy indicated for the treatment of adult patients with unresectable or …
Lynavoy
linerixibat
GlaxoSmithKline
treatment of cholestatic pruritus associated with primary biliary cholangitis (PBC) in adult patients…
Lynozyfic
linvoseltamab-gcpt
Regeneron Pharmaceuticals, Inc.
1 INDICATIONS AND USAGE LYNOZYFIC is indicated for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at l…
Triostat
Liothyronine sodium injection
SmithKline Beecham Pharmaceuticals
Treatment of myxedema coma/precoma.…