FDA-Approved Treatments
Drugs with orphan designation or indications for rare diseases — 955 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.
Glycerol Phenylbutyrate
GLYCEROL PHENYLBUTYRATE ORAL
Par Health USA, LLC
1 INDICATIONS AND USAGE Glycerol phenylbutyrate oral liquid is indicated for use as a nitrogen-binding agent for chronic management of patients with u…
Cuvposa
glycopyrrolate
Shionogi, Inc.
1 INDICATIONS AND USAGE CUVPOSA is indicated to reduce chronic severe drooling in patients aged 3 to 16 years with neurologic conditions associated wi…
SIMPONI ARIA�
golimumab
Janssen Research & Development, LLC
1 INDICATIONS AND USAGE SIMPONI ARIA is a tumor necrosis factor (TNF) blocker indicated for the treatment of: Adult patients with moderately to severe…
Vyondys 53
GOLODIRSEN
Sarepta Therapeutics, Inc.
1 INDICATIONS AND USAGE VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of th…
Halfan
Halofantrine
SmithKline Beecham Pharmaceuticals
Treatment of adults who can tolerate oral medication and who have mild to moderate malaria caused by Plasmodium falciparum or Plasmodium vivax.…
Panhematin
Hemin
Abbott Laboratories
1 INDICATIONS AND USAGE PANHEMATIN is a hemin for injection indicated for the amelioration of recurrent attacks of acute intermittent porphyria tempor…
HepaGam B
hepatitis B immune globulin (human)
Cangene Corporation
INDICATIONS AND USAGE Nabi-HB, Hepatitis B Immune Globulin (Human), is indicated for treatment of acute exposure to blood containing HBsAg, perinatal …
Supprelin LA
Histrelin
Endo Pharmaceuticals Solutions, Inc.
1 INDICATIONS AND USAGE SUPPRELIN LA (histrelin acetate) subcutaneous implant is indicated for the treatment of children with central precocious puber…
Supprelin Injection
Histrelin acetate
Roberts Pharmaceutical Corp.
1 INDICATIONS AND USAGE SUPPRELIN LA (histrelin acetate) subcutaneous implant is indicated for the treatment of children with central precocious puber…
RiaSTAP
human fibrinogen concentrate, pasteurized
CSL Behring, LLC
Treatment of acute bleeding episodes in patients with congenital fibrinogen deficiency (afibrinogenemia and hypofibrinogenemia)…
Hydrocortisone
HYDROCORTISONE
Preferred Pharmaceuticals Inc.
INDICATIONS AND USAGE Hydrocortisone Tablets are indicated in the following conditions. 1. Endocrine Disorders Primary or secondary adrenocortical ins…
Cyanokit
Hydroxocobalamin
SERB, S.A.
1 INDICATIONS AND USAGE CYANOKIT is indicated for the treatment of known or suspected cyanide poisoning. CYANOKIT is indicated for the treatment of kn…
Siklos
hydroxycarbamide (hydroxyurea)
Theravia Pharma
1 INDICATIONS AND USAGE SIKLOS ® is indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in adult and ped…
Makena
hydroxyprogesterone caproate
AMAG Pharma USA, Inc.
INDICATIONS AND USAGE Hydroxyprogesterone Caproate Injection, USP is indicated in non-pregnant women: for the treatment of advanced adenocarcinoma of …
DROXIA
HYDROXYUREA
Waylis Therapeutics LLC
1 INDICATIONS AND USAGE DROXIA is indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in patients with s…
Trogarzo
ibalizumab
Theratechnologies Inc.
1 INDICATIONS AND USAGE TROGARZO, in combination with other antiretroviral(s), is indicated for the treatment of human immunodeficiency virus type 1 (…